THE RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
GRIFFITH UNIVERSITY (Australia)
Inventor
Apicella, Michael A.
Ketterer, Margaret
Weiss, David
Edwards, Jennifer
Jennings, Michael
Jen, Freda
Abstract
Neisseria gonorrhoeaeN. gonorrhoeaeN. gonorrhoeaeNeisseria gonorrhoeaeN. gonorrhoeaeN. gonorrhoeaeN. gonorrhoeaeNeisseria gonorrhoeaeN. gonorrhoeaeN. gonorrhoeae outer membrane Phospholipase A protein and a pharmaceutically-acceptable, non-toxic vehicle, to a patient in need thereof.
C07K 14/22 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from bacteria from Neisseriaceae (F), e.g. Acinetobacter
C12N 15/11 - DNA or RNA fragmentsModified forms thereof
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
C07K 14/435 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
THE RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Ray, William
Abstract
An interactive assessment system for measuring neurologic function and method of use are provided herein. The interactive assessment system includes an interactive assessment device comprising an interactive display defining longitudinal and lateral columns of interactive nodes, each interactive node is coupled to a sensor, and has an illumination mode and a delumination mode. The interactive assessment system also including a processing device in communication with the interactive assessment device and configured to perform logic functions based upon user inputs on the interactive assessment device. The processing device includes memory wherein previously input interactions with the interactive assessment device are stored and tagged as successes, failure, or interaction events. The processing device provides instruction to the interactive assessment device to assign nodes to the illumination mode and the delumination mode.
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Loiler, Scott Allen
Abstract
Modified capsid proteins, isolated polynucleotides, methods for the preparation of modified capsid proteins, recombinant viral particles, recombinant expression systems for the generation of modified viral particles, and methods of gene editing and regulation are provided herein.
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Harper, Scott, Quenton
Taylor, Noah
Guggenbiller, Matthew
Abstract
The present invention relates to modified and improved proviral plasmid nucleic acid sequences and methods of producing AAV using these modified proviral plasmids.
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
THE TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK (USA)
Inventor
Harper, Scott Quenton
Frankel, Wayne N.
Sands, Tristan T.
Abstract
Disclosed herein are products, methods, and uses for treating, ameliorating, or delaying the progression of, and/or preventing seizures, an epileptic disease or disorder, an intellectual or developmental disability, autism, or an autism spectrum disorder associated with mutant or pathogenic Potassium Channel, Voltage Gated KQT-Like Subfamily Q, Member 3 (KCNQ3) expression. More particularly, disclosed herein are RNA interference-based products, methods, and uses for reducing or inhibiting the expression of the KCNQ3 gene and its resulting mRNA and/or protein. Even more particularly, the disclosure provides microRNA (miRNA) for reducing or inhibiting the expression of KCNQ3 and methods of using said miRNA to reduce or inhibit mutant or pathogenic KCNQ3 expression in cells and/or in cells of a subject having a genetic mutation in the KCNQ3 gene which results in disease symptoms including, but not limited to, seizures, epilepsy, intellectual and/or developmental disability, autism, or an autism spectrum disorder. Such disease symptoms, in some aspects, result from developmental and epileptic encephalopathy (DEE) attributed to various mutations in the KCNQ3 gene which result in the expression of various mutant or pathogenic forms of the KCNQ3 protein.
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
THE TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK (USA)
Inventor
Harper, Scott, Quenton
Frankel, Wayne, N.
Taylor, Noah
Abstract
[215] RNA interference-based methods and products for inhibiting the expression of pathogenic dynamin-1 (DNM1) variants and increasing the expression of DNM1 are provided. Delivery vehicles such as nanoparticles, extracellular vesicles, exosomes, or vectors, including but not limited to recombinant adeno-associated viral vectors, deliver DNAs encoding RNAs that inhibit the expression of DNM1 variants and DNAs encoding DNM1 to restore the expression of DNM1 are provided. Also provided are methods for inhibiting the expression of variant DNM1 by microRNA interference and restoring the expression of functional or normal DNM1 in cells or in human subjects by delivering a replacement DNM1 gene. The methods treat, for example, DNM1 -related disorders, such as developmental and epileptic encephalopathy (DEE), including but not limited to, Lennox-Gastaut Syndrome or infantile spasms.
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
8.
PRODUCTS AND METHODS FOR TREATING DISEASES OR DISORDERS ASSOCIATED WITH DUX4 OVEREXPRESSION
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
ARMATUS BIO, INC. (USA)
Inventor
Harper, Scott, Quenton
Wallace, Lindsay
Triplett, Michael
Price, Brian
Abstract
Disclosed herein are products, methods, and uses for treating, ameliorating, delaying the progression of, and/or preventing a muscular dystrophy or a cancer including, but not limited to, facioscapulohumeral muscular dystrophy (FSHD) or a cancer associated with DUX4 expression or overexpression. More particularly, disclosed herein are RNA interference-based products, methods, and uses for inhibiting or downregulating the expression of double homeobox 4 (DUX4). Even more particularly, the disclosure provides microRNA (miRNA) (and vectors and compositions comprising the miRNA encoding polynucleotides) for inhibiting or downregulating the expression of DUX4 and methods of using said miRNA to inhibit or downregulate DUX4 expression in cells and/or in cells of a subject having a muscular dystrophy or a cancer associated with DUX4 expression or overexpression including, but not limited to, FSHD or a cancer associated with DUX4 expression or overexpression.
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Wang, Pin-Yi
Venkataramany, Akila
Wein, Nicolas
Chandler, Dawn
Cripe, Timothy
Abstract
The disclosure relates to the field of gene therapy for the treatment of an osteosarcoma using splice-switching oligonucleotides. Increased expression of INSR-A, an isoform frequently expressed in cancer, can effectively evade current therapeutic mechanisms and contribute to resistance in cancer patients. As a means to address the ability of cancer to evade therapies through this mechanism, Applicant provides a polynucleotide comprising a promoter, for example a U7 or a U1 promoter, and a first splice-switching oligonucleotide (SSO) that targets a regulatory element of an insulin receptor (JR).
Research Institute at Nationwide Children's Hospital (USA)
Inventor
Cassady, Kevin A.
Abstract
Provided herein are non-natural herpes simplex virus (“HSV”) vectors and one or more polynucleotides encoding IL-21 or a biologically active fragment of IL-21 for use in the treatment of cancer.
THE RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Cassady, Kevin A.
Abstract
A recombinant interleukin-27 (IL27) expressing virus is described. The recombinant IL27 expressing virus comprises an oncolytic virus comprising one or more exogenous nucleic acid sequences capable of expressing in IL27 protein or a biologically active portion thereof, the exogenous nucleic acid sequences being operably linked to an expression control sequence. Methods of treating cancer by in a subject by contacting a cancer cell of the subject with a recombinant IL27 expressing virus are also described.
THE RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Maitre, Nathalie Linda
Chorna, Olena D.
Abstract
One aspect of the present disclosure include a method of utilizing an oro-motor device to activate an audio device, the method includes providing an oro-motor device having a sensor and a nipple; producing a signal when the nipple portion present in an infant's mouth when the nipple portion yields a first measured pressure over an age appropriate predetermined threshold applied to the nipple portion by the infant; responsive to the signal, playing an age appropriate audio recording for a predetermined duration on an audio device; and raising the age appropriate predetermined threshold to a raised threshold proportionally to a difference between the first measured pressure application to the nipple portion and the age appropriate predetermined threshold.
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Rodino-Klapac, Louise
Mendell, Jerry R.
Abstract
Described herein are methods of treating muscular dystrophy comprising administering a self complementary recombinant AAV (rAAV) scAAVrh74.MHCK7.hSGCB vector, methods of expressing beta-sarcoglycan gene in a patient, pharmaceutical compositions comprising the rAAV, and methods of generating the rAAV.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
A61P 21/00 - Drugs for disorders of the muscular or neuromuscular system
The Research Institute at Nationwide Children's Hospital (USA)
Inventor
Smoyer, William E.
Abstract
A method of treating nephrotic syndrome (NS) in a subject is described. The method includes administering a therapeutically effective amount of a PPARγ agonist to the subject.
A61K 31/573 - Compounds containing cyclopenta[a]hydrophenanthrene ring systemsDerivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone substituted in position 21, e.g. cortisone, dexamethasone, prednisone or aldosterone
A61K 31/4439 - Non-condensed pyridinesHydrogenated derivatives thereof containing further heterocyclic ring systems containing a five-membered ring with nitrogen as a ring hetero atom, e.g. omeprazole
A61P 13/12 - Drugs for disorders of the urinary system of the kidneys
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Martin, Paul
Abstract
The present invention relates to methods and materials for treating GNE-related disorders such as GNE myopathy, GNE-dependent ALS, thrombocytopenia, sarcopenia and aging using a dual gene recombinant adeno-associated virus comprising the GNE gene and the follistatin gene. This therapy is unique in that it can rebuild lost muscle strength at the same time that it prevents subsequent muscle disease from occurring.
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Lee, Dean
Pereira, Marcelo
Abstract
Disclosed are V-domain Ig suppressor of T cell activation (VISTA) knock-out natural killer (NK) cells and methods of making the same. Also disclosed are methods of enhancing the efficacy of NK cell killing of cancer cells by blocking VISTA expression and/or signaling.
THE RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Chandler, Dawn S.
Dominguez, Catherine E.
Abstract
A method of treating spinal muscular atrophy by inducing a heat shock response in a subject in need thereof is described. The heat shock response can be induced by heating the temperature of a tissue region of the subject above 37° C. or by administering a therapeutically effective amount of a heat shock inducing agent.
A61F 7/12 - Devices for heating or cooling internal body cavities
A61K 31/133 - Amines, e.g. amantadine having hydroxy groups, e.g. sphingosine
A61K 31/616 - Salicylic acidDerivatives thereof having the hydroxy group in position 2 esterified, e.g. salicylsulfuric acid by carboxylic acids, e.g. acetylsalicylic acid
19.
CRISPR/CAS9 BASED TREATMENT FOR PROTEIN MUTATIONS ASSOCIATED WITH MULTISYSTEM PROTEINOPATHY
C12N 15/11 - DNA or RNA fragmentsModified forms thereof
C12N 15/88 - Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using liposome vesicle
20.
Neuregulin for Protection Against Respiratory Viral Infection and Post-Viral Disease
THE RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Grayson, Mitchell
Hussain, Syed-Rehan
Abstract
A method of treating or decreasing the risk of developing a respiratory viral infection in a subject is described. The method includes administering a therapeutically effective amount of neuregulin to the subject. A method of decreasing the risk that a subject will develop post-viral airway disease by administering an effective amount of neuregulin to the subject is also described.
The Research Institute at Nationwide Children's Hospital (USA)
Inventor
Blalock, Lexie
Warren, Lauren
Lauber, Christian
Abstract
A method of gene targeting utilizing outer membrane vesicle is disclosed. As described herein outer membrane vesicles (OMVs) have the ability to modulate the expression of NRG1 intracellularly, which affects intracellular NRG1 mediated functions in addition to autocrine and paracrine signaling that support cell development, differentiation and growth. The OMVs are useful in modifying the expression of NRG1 and the expression of genes other than NRG1. The Pg OMVs also function as a gene therapy vector, as it is up taken by mammalian cells and crosses both the placental and blood brain barrier.
A61P 25/00 - Drugs for disorders of the nervous system
A61P 29/00 - Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agentsNon-steroidal antiinflammatory drugs [NSAID]
C12N 15/88 - Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using liposome vesicle
22.
PRODUCTS AND METHODS FOR INHIBITION OF EXPRESSION OF DYNAMIN-1 VARIANTS
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
THE TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK (USA)
Inventor
Harper, Scott Quenton
Frankel, Wayne N.
Abstract
RNA interference-based methods and products for inhibiting the expression of pathogenic dynamin-1 variants are provided. Delivery vehicles such as recombinant adeno-associated viruses deliver DNAs encoding RNAs that inhibit the expression of the dynamin-1 variants. The methods treat, for example, developmental and epileptic encephalopathies.
THE RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Kerlin, Bryce
Waller, Amanda
Abdelghani, Eman
Abstract
A method of treating or preventing glomerular disease or chronic kidney disease in a subject is described. The method includes administering to the subject a therapeutically effective amount of a thrombin inhibitor or a pharmaceutically acceptable salt thereof.
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
UNIVERSITY OF UTAH RESEARCH FOUNDATION (USA)
Inventor
Flanigan, Kevin
Bradbury, Allison Marie
Bonkowsky, Joshua
Pyne, Nettie Kate
Herstine, Jessica
Abstract
Provided are gene therapy vectors, such as adeno-associated virus (AAV), designed for treatment of mutations in the Eukaryotic Translation Initiation Factor 2B Subunit Epsilon (EIF2B5) gene. The EIF2B5 gene provides instructions for making one of five subunits of the elF2B protein, specifically the epsilon subunit of this protein. Such mutations are associated with a disease or disorder such as a leukoencephalopathy, a megalencephalic leukoencephalopathy, a leukodystrophy, a stroke, a migraine, epilepsy, multiple sclerosis (MS), Parkinson's disease (PD), Alzheimer's disease (AD), astrogliosis in aging, Huntington's Disease (HD), amyotrophic lateral sclerosis (ALS), Alexander disease, hepatic encephalopathy (HE), AicardinGoutieres syndrome, CLC-2-related disease, oculodentodigital dysplasia, and/or giant axonal neuropathy. Such leukoencephalopathies or leukodystrophies include, but are not limited to, Vanishing White Matter Disease (VWM). The disclosed gene therapy vectors provide a EIF2B5 cDNA to a subject in need which results in expression of a wild type or functional EIF2B5 protein. Also provided is a new promoter, designated gfa1405, which was designed to target astrocytes and neurons. Thus, compositions, nanoparticles, extracellular vesicles, exosomes, or vector comprising the gfa1405 promoter and methods of its use are also provided.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Goodman, Steven D.
Bakaletz, Lauren O.
Abstract
Provided herein are methods and compositions for disrupting biofilms in vitro and in vivo. Also disclosed are antibodies comprising a specified heavy chain (HC) immunoglobulin variable domain sequence and/or a specified light chain (LC) immunoglobulin variable domain sequence.
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Naeimi Kararoudi, Meisam
Saljoughian, Noushin
Snyder, Genesis
Lee, Dean
Abstract
Disclosed herein is a method of generating a CAR-expressing gd T cell for the use of treating cancer without the onset of Graft versus Host Disease (GvHD) thereof.
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Rodino-Klapac, Louise
Mendell, Jerry R.
Abstract
The invention provides for recombinant AAV vectors comprising a a miniaturized human micro-dystrophin gene and methods of using the recombinant vectors to reduce or prevent fibrosis in subjects suffering from muscular dystrophy.
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
A61P 19/04 - Drugs for skeletal disorders for non-specific disorders of the connective tissue
A61P 21/00 - Drugs for disorders of the muscular or neuromuscular system
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
C12N 15/11 - DNA or RNA fragmentsModified forms thereof
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
THE RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Gunn, John S.
Sandala, Jenna
Melander, Christian Corey
Woolard, Katherine June
Abstract
Compounds according to formula I: wherein Ar is an aryl or heteroaryl group, A is a C1-C3 alkyl, Z and Y are independently C1-C3 alkylene, X is C1-C3 alkylene, and R1-R5 are selected from —H, halogen, C1-C3 alkyl, or phenyl, or a pharmaceutically acceptable salt thereof, are described. The compounds can be used to decrease the amount of biofilm in a subject, and can be used together with antibacterial agents for the treatment of bacterial infection.
Compounds according to formula I: wherein Ar is an aryl or heteroaryl group, A is a C1-C3 alkyl, Z and Y are independently C1-C3 alkylene, X is C1-C3 alkylene, and R1-R5 are selected from —H, halogen, C1-C3 alkyl, or phenyl, or a pharmaceutically acceptable salt thereof, are described. The compounds can be used to decrease the amount of biofilm in a subject, and can be used together with antibacterial agents for the treatment of bacterial infection.
A61K 31/4535 - Non-condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems containing a heterocyclic ring having sulfur as a ring hetero atom, e.g. pizotifen
C07D 409/12 - Heterocyclic compounds containing two or more hetero rings, at least one ring having sulfur atoms as the only ring hetero atoms containing two hetero rings linked by a chain containing hetero atoms as chain links
29.
MATERIALS AND METHODS FOR THE TREATMENT OF LIMB GIRDLE MUSCULAR DYSTROPHY
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Flanigan, Kevin
Gushchina, Liubov V.
Abstract
Provided are gene therapy vectors, such as adeno-associated virus (AAV), designed for treatment of mutations in the TCAP gene. Such mutations are also known as telethoninopathies and are associated with such disorders as autosomal recessive limb girdle muscular dystrophy type 2G (LGMD2G), autosomal dominant dilated cardiomyopathy (DCM), hypertrophic cardiomyopathy (HCM) or idiopathic cardiomyopathy (ICM). The disclosed gene therapy vectors provide a TCAP cDNA to a subject in need which results in expression of a wild type or functional TCAP or telethonin protein.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
A61K 9/00 - Medicinal preparations characterised by special physical form
A61K 31/453 - Non-condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems containing a six-membered ring with oxygen as a ring hetero atom
A61K 31/56 - Compounds containing cyclopenta[a]hydrophenanthrene ring systemsDerivatives thereof, e.g. steroids
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
LUDWIG INSTITUTE FOR CANCER RESEARCH (Switzerland)
Inventor
Kaspar, Brian K.
Foust, Kevin
Cleveland, Don W.
Abstract
The present invention relates to RNA-based methods for inhibiting the expression of the superoxide dismutase 1 (SOD-1) gene. Recombinant adeno-associated viruses of the invention deliver DNAs encoding RNAs that knock down the expression of SOD-1. The methods have application in the treatment of amyotrophic lateral sclerosis.
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
A61K 9/00 - Medicinal preparations characterised by special physical form
A61K 31/7105 - Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
C12N 7/00 - Viruses, e.g. bacteriophagesCompositions thereofPreparation or purification thereof
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Bakaletz, Lauren O.
Goodman, Steven D.
Abstract
Disclosed are approaches to determining a sensitivity of a bacterium to a given antibiotic and generating targeted treatments based on the sensitivity. One or more antibiotics may be selected for a chronic/recurrent infection resulting from a biofilm so as to reduce dose and or length of course of antibiotic treatment. For example, if a bacterial pathogen is determined to be sensitive to an antibiotic in its planktonic form but resistant to that antibiotic in its biofilm form, then the biofilm may be dispersed or disrupted from the biofilm residence in order to clear the infection. In various embodiments, a dispersal or disruption method and/or agent may be determined based at least in part on a rate of bacterial release from a biofilm that sensitizes the pathogen to a chosen antibiotic.
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Saad, Nizar
Abstract
Products, methods, and uses for treating, ameliorating, delaying the progression ui, and/or preventing a disease or disorder associated with expression of an aberrant lamin A (LMNA) gene or progerin gene are provided. Such disease or disorder includes, but is not limited to, a laminopathy, progeroid syndrome, progeria, or aging disorder resulting from the aberrant expression of LMNA or progerin. In some instances, the progeria is Hutchinson- Gilford progeria syndrome (HOPS). In some instances, the disease or disorder associated with the expression of progerin is premature aging or natural aging including, but not limited to, atherosclerosis, alopecia, osteoporosis, cardiovascular disease, skin abnormalities, fat storage, stroke, myocardial infarction, stroke, heart failure, muscle wasting, muscle weakness, myotonia, skeletal muscle problems, abnormalities of the retina, hip weakness, abdominal muscle weakness, joint and spinal abnormalities, lower leg weakness, shoulder weakness, hearing loss, and/or tissue inflammation. More particularly, disclosed herein are RNA interference-based products, methods, and uses for inhibiting or downregulating the expression of progerin. Even more particularly, the disclosure provides microRNA (miRNA) for inhibiting or downregulating the expression of progerin and methods of using said miRNA to inhibit or downregulate progerin expression in cells and/or in cells of a subject having a condition resulting from the expression of progerin including, but not limited to, HGPS or progeria, an HGPS-like condition affecting LMNA mutations that affect exon 11 splicing, or a condition resulting from the expression of progerin.
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Rodino-Klapac, Louise
Griffin, Danielle
Mendell, Jerry R.
Abstract
Described herein are methods of treating muscular dystrophy in a subject, comprising administration of a recombinant AAV vector AAVrh74.tMCK.SGCA using a systemic route of administration and at a dose of about 1.0×1012 vg/kg to about 5.0×1015 vg/kg. Further disclosed are methods of expressing alpha-sarcoglycan gene in a cell or in a subject in need thereof, decreasing a serum CK level, and increasing alpha-sarcoglycan positive fibers in muscle tissue of a subject.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
C07H 21/04 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
THE RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Heater, Thomas
Coles, Mary
Abstract
A tracheostomy trainer includes a head pivotally connected to a neck and a chest, he head, neck and chest define an interior space, and the neck defines a tracheostomy opening. The trainer further includes a tube entrance coupled to a mouth, an upper airway tube fluidly coupling a chest tube to the tube entrance, the upper airway tube residing at least partially in the head and at least partially in the neck, and the chest tube fluidly coupling the upper airway tube to a lung bag. The chest tube resides at least partially within the chest, and the lung bag resides within the chest. At least one of the upper airway tube or the chest tube defines a trach tube opening, the trach tube opening aligning with the tracheostomy opening, the upper airway tube, the chest tube, and the lung bag comprising an air tight fluid path, wherein fluid enters and exits the air tight fluid path via the mouth and the trach tube opening.
C12Q 1/00 - Measuring or testing processes involving enzymes, nucleic acids or microorganismsCompositions thereforProcesses of preparing such compositions
THE RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Tulchin-Francis, Kirsten
Romer, Holly
Richard, Heather
Kadado, Allen
Abstract
A brace compliance system and method of use are described herein. The brace compliance system includes a brace compliance device comprising a plurality of sensors, a brace compliance presentation device having a screen to display image and is accessible by a care provider. The brace compliance system further includes a processing device in communication with the brace compliance device and the brace compliance presentation device. The processing device receives sensor data from the plurality of sensors of the brace compliance device indicating brace wear time, wherein responsive to receiving brace wear time, the processing device determines if the brace wear time is below a provider provided threshold, and presents a notification to the brace compliance presentation device.
A61B 5/00 - Measuring for diagnostic purposes Identification of persons
A61F 5/01 - Orthopaedic devices, e.g. long-term immobilising or pressure directing devices for treating broken or deformed bones such as splints, casts or braces
A63B 24/00 - Electric or electronic controls for exercising apparatus of groups
37.
METHODS OF QUANTIFYING SKELETAL MUSCLE PERFUSION USING PET IMAGING
Research Institute at Nationwide Children’s Hospital (USA)
Inventor
Stacy, Mitchel
Chou, Ting-Heng
Abstract
Described herein are methods for quantifying skeletal muscle perfusion, skeletal muscle metabolism, and active vascular calcification by positron emission tomography (PET) imaging of labeled radionuclides, such as 18F-labeled radionuclide agents.
A61B 6/50 - Apparatus or devices for radiation diagnosisApparatus or devices for radiation diagnosis combined with radiation therapy equipment specially adapted for specific body partsApparatus or devices for radiation diagnosisApparatus or devices for radiation diagnosis combined with radiation therapy equipment specially adapted for specific clinical applications
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Zhao, Mingtao
Garg, Vidu
Texter, Karen
Alonzo, Matthew
Abstract
Provided are noninvasive methods for detecting a single ventricle heart defect (SVHD) in an unborn fetus. Also provided are methods of diagnosing the risk of SVHD in a fetus, and methods for treating SVHD. Finally, kits and systems for detecting SVHD are disclosed as well.
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Martin, Paul Taylor
Abstract
The present disclosure relates to recombinant adeno-associated virus (rAAV) delivery of a GALGT2 polynucleotide. The disclosure provides rAAV and methods of using the rAAV for GALGT2 gene therapy of neuromuscular disorders. Exemplary neuromuscular disorders include, but are not limited to, muscular dystrophies such as Duchenne muscular dystrophy, Congenital Muscular Dystrophy 1A and Limb Girdle Muscular Dystrophy 2D.
A61P 21/00 - Drugs for disorders of the muscular or neuromuscular system
C07H 21/04 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
Research Institute at Nationwide Children's Hospital (USA)
Inventor
Harper, Scott Quenton
Abstract
The present invention relates to a tissue-specific promoter system for expressing microRNA (miRNA) for RNA interference-based methods of gene therapy. In these systems, the miRNA will inhibit gene expression or replace natural miRNA expression using microRNA.
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
A61K 9/00 - Medicinal preparations characterised by special physical form
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
A61P 21/00 - Drugs for disorders of the muscular or neuromuscular system
C07H 21/02 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
C12N 7/00 - Viruses, e.g. bacteriophagesCompositions thereofPreparation or purification thereof
C12Q 1/70 - Measuring or testing processes involving enzymes, nucleic acids or microorganismsCompositions thereforProcesses of preparing such compositions involving virus or bacteriophage
42.
SYSTEMS AND METHODS FOR OPTIMIZED PATIENT SPECIFIC TISSUE ENGINEERING VASCULAR GRAFTS
Research Institute at Nationwide Children's Hospital (USA)
Inventor
Breuer, Christopher
Strouse, Robert
Ung-Lee, Yong
Best, Cameron
Hibino, Narutoshi
Abstract
It has been established that optimizing cell seeding onto tissue engineering vascular grafts (TEVG) is associated with reduced inflammatory responses and reduced post-operative stenosis of TEVG. Cell seeding increased TEVG patency in a dose dependent manner, and TEVG patency improved when more cells were seeded, however duration of incubation time showed minimal effect on TEVG patency. Methods of engineering patient specific TEVG including optimal numbers of cells to maintain graft patency and reduce post-operative stenosis are provided. Closed, single-use customizable systems for seeding TEVG are also provided. Preferably the systems are custom-designed based on morphology of the patient specific graft, to enhance the efficacy of cell seeding.
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Meyer, Kathrin Christine
Bradbury, Allison Marie
Likhite, Shibi
Abstract
The present disclosure relates to methods of treating conditions associated with a need for the electrogenic sodium- and chloride-coupled y-aminobutyric acid transporter (GAT-1) protein. for example due to a defective SLC6A1 gene as in pediatric epileptic encephalography. In particular. the disclosure provides gene therapy vectors to specifically treat loss of expression of the GAT-1 protein and/or reduced GAT-1 protein levels.
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
44.
MATERIALS AND METHODS FOR THE TREATMENT OF NEUROFIBROMIN 1 MUTATIONS AND DISEASES RESULTING THEREFROM
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
UNIVERSITY OF MASSACHUSETTS (USA)
Inventor
Bradbury, Allison, Marie
Sena-Esteves, Miguel
Abstract
Provided are gene therapy vectors, such as adeno-associated virus (AAV), designed for treatment of mutations in the neurofibromin 1 (NF1) gene. The disclosed gene therapy vectors provide a mini-NF1 cDNA to a subject in need which results in expression of a functional NF1 protein. Also provided are compositions, nanoparticles, extracellular vesicles, exosomes, or vector comprising the NF1 gene with nerve-cell specific and Schwann-cell specific promoters and methods of using the mini-NF1 gene with nerve-cell specific and Schwann-cell specific promoters in treating neurofibromatosis type 1. Also provided are novel mini-NF1 gene constructs.
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Martin, Paul, Taylor
Abstract
The disclosure provides gene therapy vectors, such as adeno-associated virus (AAV), designed for treatment of Lysosomal Acid Lipase Deficiency (LAL-D) disorders, such as Wolman disease and cholesteryl ester storage disease (CESD), nonalcoholic fatty liver disease (NAFLD), or nonalcoholic steatohepatitis (NASH). The disclosed rAAV provide a wild type lipase A (LIPA) cDNA to a subject in need which results in expression of the wild type protein.
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN' S HOSPITAL (USA)
Inventor
Harper, Scott Quenton
Rashnonejad, Afrooz
Abstract
RNA interference-based products and methods for inhibiting the expression of a mutant myelin protein zero (MPZ) gene in a cell or in the cells of a subject are disclosed. The disclosure includes microRNA that specifically target various regions of the MPZ gene to knock down expression of the aberrant protein. Additionally, the disclosure includes delivery of a nucleic acid encoding normal, wild-type, or functionally active MPZ protein. Additionally, the disclosure includes recombinant adeno-associated viruses to deliver nucleic acids encoding the microRNAs to knock down the expression of aberrant MPZ protein and/or to deliver nucleic acids encoding normal, wild-type, or functionally active MPZ protein. The disclosure includes methods of using these nucleic acids in the treatment of diseases associated with MPZ gene mutations including, but not limited to, Charcot-Marie-Tooth disease type 1B (CMT 1B) disease.
The Research Institute at Nationwide Children's Hospital (USA)
Inventor
Maitre, Nathalie
Ray, Will
Chorna, Olena
Evans, Ellyn
Abstract
A sanitizable audio device for use in neonatal care and method of use are provided herein. The audio device comprises a smooth shell defining an outer surface and an inner surface. The outer surface forms a curved continuous smooth surface. The inner surface supports electronic elements. The shell defines one or more switch apertures, housing one or more actuatable electrical switches, in communication with the electronic elements. The method of use comprises generating an audio output including selecting content that is age appropriate for a particular infant, altering a sound emission of a recording of a care-giver of the particular infant, and programming a music player to emit the audio output below an age dependent volume, for an age dependent number of intervals per day and per week, for an age dependent duration.
G08B 21/02 - Alarms for ensuring the safety of persons
G08B 3/10 - Audible signalling systemsAudible personal calling systems using electric transmissionAudible signalling systemsAudible personal calling systems using electromagnetic transmission
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Lee, Dean Anthony
Naeimi Kararoudi, Meisam
Abstract
Disclosed are single-chain variable fragments, chimeric antigen receptors, and methods to treat and prevent CD33-associated pathologies, such as hematologic cancers and neurological disorders.
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
Research Institute at Nationwide Children's Hospital (USA)
Inventor
Sahenk, Zarife
Abstract
The present disclosure relates to recombinant adeno-associated virus (rAAV) delivery of a neurotrophin 3 (NT-3) polynucleotide. The disclosure provides rAAV and methods of using the rAAV for NT-3 gene therapy to improve muscle strength, stimulate muscle growth and to treat muscle wasting disorders, such as muscular dystrophy and Charcot-Marie-Tooth neuropathy.
THE RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
GEORGIA TECH RESEARCH CORPORATION (USA)
Inventor
Krishnamurthy, Rajesh
Dasi, Lakshmi Prasad
Abstract
A computer-implemented method for predicting risk of ischemia in anomalous aortic origin of a coronary artery (AAOCA) includes receiving medical imaging data of a patient. The method includes extracting, from the medical imaging data, patient-specific morphological imaging biomarkers pertaining to AAOCA and incorporating the biomarkers into a computer model. The method includes simulating, using the computer model, hemodynamics of the patient under simulated stress conditions and a combination of variables, the variables comprising physiological properties of the patient. The method also includes predicting and outputting a patient-specific risk profile of ischemia and/or sudden cardiac death based on simulated hemodynamics.
G16H 50/50 - ICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for simulation or modelling of medical disorders
G16H 10/60 - ICT specially adapted for the handling or processing of patient-related medical or healthcare data for patient-specific data, e.g. for electronic patient records
G16H 20/00 - ICT specially adapted for therapies or health-improving plans, e.g. for handling prescriptions, for steering therapy or for monitoring patient compliance
G16H 30/20 - ICT specially adapted for the handling or processing of medical images for handling medical images, e.g. DICOM, HL7 or PACS
G16H 50/30 - ICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for calculating health indicesICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for individual health risk assessment
G16H 50/70 - ICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for mining of medical data, e.g. analysing previous cases of other patients
G16H 70/20 - ICT specially adapted for the handling or processing of medical references relating to practices or guidelines
53.
PROTEIN ENGINEERING MICRO-TUBERIN GENE THERAPY CANDIDATES FOR TUBEROUS SCLEROSIS COMPLEX TYPE 2
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
OHIO STATE INNOVATION FOUNDATION (USA)
Inventor
Hester, Mark
Mcelroy, Craig
Abstract
This disclosure is directed to engineered micro-Tuberin constructs that can be used in gene therapy for treating tuberin deficiency/mTOR hyperactivation. The disclosed engineered micro-Tuberins are smaller than wild type Tuberin and retain all important domains and functions of the wild type Tuberin protein. Also disclosed are methods for delivering the engineered micro-Tuberins and methods for treating Tuberin deficiency/mTOR hyperactivation.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Harper, Scott Quenton
Guggenbiller, Matthew
Taylor, Noah
Abstract
Disclosed herein is a modular system, i.e., a DNA expression cassette, specifically designed to convert therapeutic miRNA expression cassettes from the use of ubiquitous RNA polymerase Ill-based promoters to the use of RNA polymerase Il-based promoters to allow for tissue specific expression of the miRNAs while maintaining fidelity and efficacy of processing. Also disclosed herein are compositions, vectors, nanoparticles, extracellular vesicles, and exosomes comprising the nucleic acid expression cassette, as well as methods of use and methods of treatment comprising the DNA expression cassette.
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Sahenk, Zarife
Abstract
Provided are gene therapy vectors, such as adeno-associated virus (AAV), compositions and methods for treatment of diseases caused by misfolded proteins. The disclosed rAAV comprise a nucleotide sequence encoding the BCL2 Associated Athanogene 3 (BAG-3) protein, and methods of administering these rAAV to treat of diseases and disorders associated with protein misfolding and/or aggregation in o a subject in need, which results in increased targeting of aggregation prone proteins for degradation through the BAG3-mediated selective macroautophagy pathway, thereby restoring proteostasis. Thedisclosed gene therapy vectors, such as rAAV constructs also are used for treatment of inclusion body myositis (IBM) associated with Paget disease of bone and frontotemporal dementia (IBMPFD) and multisystem proteinopathy.
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
The Research Institute at Nationwide Children's Hospital (USA)
Inventor
Kaspar, Brian K.
Abstract
The present invention is directed to methods and materials for producing recombinant viruses. In particular, methods and materials are provided for producing recombinant viruses in eukaryotic microalgae such as Chlamydomonas reinhardtii. Recombinant adeno-associated viruses are examples of recombinant viruses produced according to the invention.
The Research Institute at Nationwide Children's Hospital (USA)
Inventor
Song, Xiaotong
Wang, Ruoning
Sarkar, Abhijit
Hu, Yue
Abstract
The present application provides methods and compositions for treating cancers using a CAR T cell therapy platform. Also provided are methods and use of the CAR T cells for treating diseases and conditions, such as cancer, and in particular any disease or condition associated with elevated adenosine or other associate marker.
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Vaidyanathan, Sriram
Abstract
This disclosure provides gene-editing systems and compositions that comprise modified single stranded homology directed repair (HDR) template polynucleotides that improve gene editing. Also provided are methods for using the disclosed gene-editing systems and compositions.
Research Institute At Nationwide Children's Hospital (USA)
Ludwig Institute For Cancer Research (Switzerland)
Inventor
Kaspar, Brian K.
Foust, Kevin
Cleveland, Don W.
Abstract
The present invention relates to RNA-based methods for inhibiting the expression of the superoxide diamutase 1 (SOD-1) gene. Recombinant adeno-associated viruses of the invention deliver DNAs encoding RNAs that knock down the expression of SOD-1. The methods have application in the treatment of amyotrophic lateral sclerosis.
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
A61K 9/00 - Medicinal preparations characterised by special physical form
A61K 31/7105 - Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
C12N 7/00 - Viruses, e.g. bacteriophagesCompositions thereofPreparation or purification thereof
THE RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Hoang, Ky Van
Gunn, John S.
Melander, Christopher
Sorge, Amy
Woolard, Katherine
Carpenter, Morgan
Abstract
A method of treating or preventing infection by an intracellular pathogen in a subject is described. The method includes administering to the subject a therapeutically effective amount of a composition including KH-1, KH-2, or a derivative and/or a pharmaceutically acceptable salt thereof. A method of treating or preventing bacterial inflammation in a subject is also described. New KH-1 and KH-2 derivatives are also described.
A61K 31/407 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil condensed with heterocyclic ring systems, e.g. ketorolac, physostigmine
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
OHIO STATE INNOVATION FOUNDATION (USA)
Inventor
Peeples, Mark
Li, Jianrong
Lu, Mijia
Zhang, Yuexiu
Chamblee, Michelle
K C, Mahesh
Abstract
Provided here a recombinant vesicular stomatitis virus (rVSV) vectors that comprise a coronavirus prefusion spike (preS) protein, where the preS protein is from a SARS-CoV-1, SARS-CoV-2, or Middle East Respiratory Syndrome (MERS-CoV). The prefusion spike proteins are mutated to express 6 prolines in place of their native S sequences to prevent cleavage and to make the preS protein more immunogenic than an S protein lacking those mutations. Compositions comprising the rVSV encoding preS proteins and methods of their use are provided. Also provided are methods and compositions for producing isolated IgY antibodies against the 6-proline preS proteins and methods of using the isolated IgY antibodies for treatment and prophylaxis to a coronavirus exposure or infection.
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN’S HOSPITAL (USA)
Inventor
Harper, Scott Quenton
Liu, Jian
Coppens, Sara
Wallace, Lindsay
Abstract
The present invention relates to RNA interference-based methods for inhibiting the expression of the DUX4 gene, a double homeobox gene on human chromosome 4q35. Recombinant adeno-associated viruses of the invention deliver DNAs encoding microRNAs that knock down the expression of DUX4. The methods have application in the treatment of muscular dystrophies such as facioscapulohumeral muscular dystrophy.
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
The Cyprus Foundation for Muscular Dystrophy Research D/B/A The Cyprus Institute of Neurology... (Cyprus)
Inventor
Harper, Scott Quenton
Kleopa, Kleopas
Stavrou, Marina
Abstract
RNA interference-based methods and products for inhibiting the expression of a peripheral myelin protein-22 gene are provided. RNAs that inhibit the peripheral myelin protein-22 gene are provided as well as DMAs encoding the RNAs. Delivery vehicles such as recombinant adeno-associated viruses deliver DMAs encoding RNAs that inhibit the peripheral myelin protein-22 gene. The methods treat Charcot-Marie-Tooth Disease such as Charcot-Marie-Tooth Disease Type 1 A (CMT1A).
The Reasearch Institute at Nationwide Children's Hospital (USA)
Inventor
Malhotra, Prashant Solanki
Huefner, Janelle
Luna, John
Satyapriya, Anand
Lucius, Shana Nicole
Abstract
An interactive reading assistance system for assisting hearing impaired users read including an interactive reading assistance device comprising an interactive display defining a touch screen area is presented herein. The interactive reading assistance device includes a processing device having a memory and a processor configured to perform logic functions based upon user inputs on the interactive reading assistance device. One or more texts are parsed by the processing device into text segments, assigned tags, and stored in the memory. The interactive reading assistance device presents the one or more texts to a reader in a recording mode. The interactive reading assistance device presents a prompt to the reader to read and record the text segments identified based upon input therapeutic goals based upon the assigned tags. The recorded text segments are in memory. The recorded text segments are presented as associated with the respective text segments present in the one or more texts.
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Mihi, Belgacem
Besner, Gail E.
Abstract
A miniaturized physiological mimicry platform is disclosed. The platform may include microfluidic chips and other microphysiological devices and their use to emulate the anatomies and/or functions of tissues, such as single and multiple organ systems. Various manufacturing approaches are disclosed for fabricating chips of different sizes. Formulations of extracellular matrices that better sustain attachment and expansion of different cell types various devices are also provided.
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Lee, Dean Anthony
De Souza Fernandes Pereira, Marcelo
Abstract
The present disclosure relates to combination anti-cancer therapies using a transforming growth factor-beta (TGF-β) Superfamily-Imprinted Natural Killer (TGF-βi NK) cell and CD38-targeting agents and methods of use thereof.
A61K 35/17 - LymphocytesB-cellsT-cellsNatural killer cellsInterferon-activated or cytokine-activated lymphocytes
A61K 39/395 - AntibodiesImmunoglobulinsImmune serum, e.g. antilymphocytic serum
C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
C12N 5/0783 - T cellsNK cellsProgenitors of T or NK cells
G01N 33/554 - ImmunoassayBiospecific binding assayMaterials therefor with an insoluble carrier for immobilising immunochemicals the carrier being a biological cell or cell fragment, e.g. bacteria, yeast cells
68.
METHODS FOR TREATING FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY (FSHD)
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Harper, Scott Quenton
Eidahl, Jocelyn
Wallace, Lindsay
Knox, Renatta
Abstract
Disclosed herein are methods and uses for treating, ameliorating, delaying the progression of, and/or preventing a muscular dystrophy or a cancer including, but not limited to, facioscapulohumeral muscular dystrophy (FSHD) or a sarcoma. More particularly, disclosed herein are methods of using small molecule protein arginine methylation (PRMT) inhibitors, and uses of these inhibitors, for inhibiting methylation of amino acids, e.g., arginine, in the double homeobox 4 (DUX4) protein. Even more particularly, the disclosure provides methods of using such methylation inhibitors or arginine methylation inhibitors for inhibiting methylation of the DUX4 protein resulting in reduced DUX4-activated cell death, including reduced DUX4-activated muscle cell death and/or reduced DUX4 target gene activation. The disclosure provides, in some aspects, methods of using protein methylation inhibitors including, but not limited to salvianolic acid A (SAA), or a derivative thereof, or adenosine dialdehyde (ADOX), or a derivative thereof for inhibiting methylation of arginine residues of the DUX4 protein in cells in vitro, ex vivo, or in vivo in the cells of a subject at risk of or suffering from a muscular dystrophy or a cancer associated with DUX4 overexpression.
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Lee, Dean Anthony
De Souza Fernandes Pereira, Marcelo
Cash, Catherine Alexandra
Abstract
The aryl hydrocarbon receptor nuclear translocator (ARNT) binds to ligand-bound aryl hydrocarbon receptor and is a co-factor for transcriptional regulation by hypoxia-inducible factor 1 alpha.Both the AHR and HIF-1 alpha proteins are important sensors related to immune suppression in the tumor environment. The present disclosure relates to ARNTKO natural killer cells and methods of use thereof in cancer therapy.
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Flanigan, Kevin
Wein, Nicolas Sebastien
Simmons, Tabatha
Abstract
Products and methods for treating or preventing muscular dystrophies in patients with duplications of exon (2) in their DMD gene or DMD mutations of any class that maintain a functional IRES sequence within exon (5), and an open reading frame from exon (6) though the end of the DMD gene are provided. Gene therapy vectors, such as adeno-associated virus (AAV) vectors and methods of using these vectors to express DMD are provided. The products and methods are used for treating and/or preventing muscular dystrophies, such as Duchenne Muscular Dystrophy or Becker Muscular Dystrophy.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
A61K 9/00 - Medicinal preparations characterised by special physical form
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
A61P 21/00 - Drugs for disorders of the muscular or neuromuscular system
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Flanigan, Kevin
Stephenson, Anthony Aaron
Abstract
The disclosure relates to the field of gene therapy for the treatment of a muscular dystrophy including, but not limited to, Duchenne's muscular dystrophy (DMD), Becker's muscular dystrophy (BMD), or intermediate muscular dystrophy (IMD). More particularly, the disclosure provides nucleic acids, including nucleic acids comprising guide RNAs (gRNAs) and nucleic acids encoding gRNAs to be used with nucleic acids encoding clustered regularly-interspaced short palindromic repeat associated protein 9 (Cas9), and adeno-associated virus (AAV) comprising the nucleic acids to deliver nucleic acids encoding guide RNAs and Cas9 to correct single or multiple DMD exon duplication mutations for use in treating a muscular dystrophy including, but not limited to, DMD, BMD, or IMD, resulting from an exon duplication mutation amenable to CRISPR-Cas9 therapy of the DMD gene.
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Martin, Paul Taylor
Abstract
Products and methods for treating dystroglycanopathies and laminin-deficient muscular dystrophies are provided. In the methods, a protein including a linker domain, such as the heparin-binding domain of Heparin-Binding Epidermal Growth Factor-Like Growth Factor (HBEGF), is delivered to patients.
C07K 14/485 - Epidermal growth factor [EGF], i.e. urogastrone
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
A61P 21/00 - Drugs for disorders of the muscular or neuromuscular system
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
C07K 14/78 - Connective tissue peptides, e.g. collagen, elastin, laminin, fibronectin, vitronectin or cold insoluble globulin [CIG]
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Lee, Dean Anthony
Naeimi Kararoudi, Meisam
De Souza Fernandes Pereira, Marcelo
Abstract
The present disclosure relates methods of engineering natural killer cells for treating, preventing, inhibiting, decreasing, and/or ameliorating diseases, including but not limited to cancer and other proliferative diseases.
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Meyer, Kathrin
Likhite, Shibi
Powers, Samantha, Lynn
Abstract
The present disclosure relates to methods of modulating transcription of target genes using transcription modulators termed RNA-based transcription modulators or "RPMs". The RPMs attract transcription regulators to the genes.
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
A61P 21/02 - Muscle relaxants, e.g. for tetanus or cramps
C07H 21/00 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
C12N 15/63 - Introduction of foreign genetic material using vectorsVectorsUse of hosts thereforRegulation of expression
The Research Institute at Nationwide Children' s Hospital (USA)
Inventor
Maitre, Nathalie
Jeanvoine, Amaud
Abstract
A movement assessment system and method of use are described herein. The movement assessment system includes a movement assessment device comprising a plurality of sensors, a movement assessment presentation device having a screen to display image; and a processing device in communication with the movement assessment device and the movement assessment presentation device. The processing device receives displacement data from the movement assessment device. Responsive to receiving the displacement data, the processing device identifies features from displacement data including at least one of motion, amplitude and speed variation of sensed motion, extracts a spectrum from the features to identify feature variability over time, identifies from spectrum potential disease based upon a percentage of abnormal movement over a likelihood threshold being identified, and presents the potential disease to user on the movement assessment presentation device.
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Flanigan, Kevin
Wein, Nicolas Sebastien
Simmons, Tabatha
Vulin-Chaffiol, Adeline
Abstract
Products and methods for treating or preventing muscular dystrophies in patients with mutations in the 5′ end of their DMD gene are provided. In some aspects, oligonucleotides, antisense phosphorodiamidate morpholino oligomers (PMO), and antisense cell penetrating peptide-conjugated PMOs (PPMOs) are provided for skipping exon 2 of the DMD gene. These oligonucleotides and oligomers can selectively suppress mutant forms of the dystrophin protein while allowing a functional form of the dystrophin protein to be expressed in sufficient quantity to retain its function in the cell. The oligonucleotides or oligomers can regulate or restore expression of transcripts of the DMD gene and a functional form of the dystrophin protein. Methods comprising administering the oligonucleotides. PMO, and PPMO targeting the DMD gene are provided. The products and methods are used for treating, ameliorating and/or preventing muscular dystrophies, such as Duchenne Muscular Dystrophy or Becker Muscular Dystrophy.
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
A61K 31/573 - Compounds containing cyclopenta[a]hydrophenanthrene ring systemsDerivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone substituted in position 21, e.g. cortisone, dexamethasone, prednisone or aldosterone
A61P 21/00 - Drugs for disorders of the muscular or neuromuscular system
77.
PRODUCTS AND METHODS FOR TREATING MUSCULAR DYSTROPHY
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Wein, Nicolas Sebastien
Flanigan, Kevin
Abstract
Products and methods for treating or preventing muscular dystrophies in patients with mutations in any of exons 6, 7, 8, or 9 in their DMD gene are provided. Gene therapy vectors, such as adeno-associated virus (AAV) vectors, and methods of using these vectors to deliver nucleic acids comprising DMD antisense sequences in regulating or restoring expression of transcripts of the DMD gene and a functional form of the dystrophin protein are provided. The products and methods are used for treating, ameliorating and/or preventing muscular dystrophies, such as Duchenne Muscular Dystrophy or Becker Muscular Dystrophy.
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
A61K 9/00 - Medicinal preparations characterised by special physical form
A61K 31/573 - Compounds containing cyclopenta[a]hydrophenanthrene ring systemsDerivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone substituted in position 21, e.g. cortisone, dexamethasone, prednisone or aldosterone
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
A61P 21/00 - Drugs for disorders of the muscular or neuromuscular system
THE RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
DANA-FARBER CANCER INSTITUTE, INC. (USA)
Inventor
Stanton, Benjamin
Sunkel, Benjamin Douglas
Qi, Jun
Abstract
A method of treating rhabdomyosarcoma in a subject is described that includes administering a therapeutically effective amount of a PROTAC compound that results in the degradation of FOXO1 or a PAX3-FOXO1 fusion protein. A method of studying the chromatin-level effects of switching defective and sucrose nonfermenting (SWI/SNF) inactivators, comprising degrading ATPase using a PROTAC compound, and measuring the biological effect of the loss of SWI/SNF complexes is also described.
C07D 403/14 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group containing three or more hetero rings
C12Q 1/68 - Measuring or testing processes involving enzymes, nucleic acids or microorganismsCompositions thereforProcesses of preparing such compositions involving nucleic acids
79.
EVALUATION OF PATIENTS WITH CYSTIC FIBROSIS USING SWEAT
THE RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Hayes, Don
Woodley, Fred
Kopp, Ben
Abstract
A method of determining if a patient having cystic fibrosis has an increased risk of having or developing pulmonary exacerbations is described. The method includes determining the level of one or more metabolites associated with pulmonary exacerbations in a sweat sample from the patient, and characterizing the patient as having an increased risk of having or developing pulmonary exacerbations if one or more metabolites associated with pulmonary exacerbation are significantly different from a control value. A method of evaluating the response of a patient having pulmonary exacerbations to treatment is also described.
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Meyer, Kathrin Christine
Dennys-Rivers, Cassandra Nicole
Abstract
Methods and materials for treating Pitt Hopkins Syndrome comprising intrathecal delivery of recombinant Adeno-associated virus 9 (rAAV9) encoding Methyl-CpG binding protein 2 (MECP2) are provided.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
A61P 25/00 - Drugs for disorders of the nervous system
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Chang, Long-Sheng
Flanigan, Kevin
Likhite, Shibi
Meyer, Kathrin
Abstract
The present disclosure relates to methods of treating conditions associated with a need for Merlin protein, for example due to a defective Neurofibromin 2/Merlin (NF2) gene as in neurofibromatosis type 2 (NF2). In particular. the disclosure provides gene therapy vectors to specifically treat loss of expression of the Merlin protein or reduced Merlin protein levels.
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Lee, Dean
Naeimi Kararoudi, Meisam
Abstract
The present disclosure provides plasmids, nucleic acids, or constructs for use with a CRISPR/CAS9 system to genetically engineer T cells. In some aspects, disclosed herein are method of using such engineering T cells for treating cancers.
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Rodino-Klapac, Louise
Abstract
The invention provides for AAV vectors expressing the ANO5 gene and antioxidant therapy as methods of inducing muscle regeneration and a method of treating muscular dystrophy.
A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
A61P 21/00 - Drugs for disorders of the muscular or neuromuscular system
C07K 14/435 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
A61K 35/17 - LymphocytesB-cellsT-cellsNatural killer cellsInterferon-activated or cytokine-activated lymphocytes
C07K 16/30 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants from tumour cells
86.
ADENO-ASSOCIATED VIRUS VECTOR DELIVERY OF B-SARCOGLYCAN AND MICRORNA-29 AND THE TREATMENT OF MUSCULAR DYSTROPHY
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Rodino-Klapac, Louise
Mendell, Jerry R.
Abstract
Described herein are recombinant AAV vectors comprising a polynucleotide sequence comprising β-sarcoglycan and methods of using the recombinant vectors to reduce or prevent fibrosis in a mammalian subject suffering from a muscular dystrophy. Also described herein are combination therapies comprising administering AAV vectors(s) expressing β-sarcoglycan and miR-29c to a mammalian subject suffering from a muscular dystrophy.
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
A61P 21/00 - Drugs for disorders of the muscular or neuromuscular system
A61P 25/14 - Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
THE RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Jadcherla, Sudarshan
Abstract
An oral regulation system and method of use are described herein. The oral regulation system includes an oral regulation device, an oral regulation presentation device, and a processing device. The stimulation device defining a stimulation opening and configured to be coupled to a stimulation administrator, and having a valve for fluidly coupling the stimulation opening to the stimulation administrator; and one or more sensors fluidly coupled to the valve for receiving stimulation information generated in response to stimulation provided by the stimulation administrator. The oral regulation presentation device having a screen to display images, the processing device in communication with the oral regulation device and the oral regulation presentation device.
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Barker, Jenny
Blum, Kevin
Abstract
Disclosed are methods for treating or preventing capsular contracture or capsule formation in a subject with an implant, the method comprising administering to the subject tamoxifen or a metabolite thereof in a localized form. Also disclosed is a surgical implant coated with tamoxifen or a metabolite thereof.
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Goodman, Steven D.
Bakaletz, Lauren O.
Abstract
Provided herein are methods for preventing or treating an infection caused by a Nontuberculous mycobacterium (NTM) species in a subject comprising administering to the subject an effective amount of an antibody or an antigen-binding fragment thereof that binds to a tip region of a DNABII peptide. Also provided in are methods for sensitizing a biofilm to an antibiotic agent, wherein the biofilm comprises a Nontuberculous mycobacterium (NTM) species, the method comprising contacting the biofilm with an antibody or an antigen¬ binding fragment thereof that binds to a tip region of a DNABII peptide.
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Hoelzle, David
Adunka, Oliver F.
Wiet, Gregory
Abstract
A robotic system for cochlear implantation (CI) is described herein. The system includes a robotic tool configured to hold a cochlear implant electrode array and a controller that is operably coupled to the robotic tool. The controller includes a processor and a memory. The memory has computer-executable instructions stored thereon that, when executed by the processor, cause the processor to control the robotic tool with the degrees of freedom of a human hand.
A61B 90/00 - Instruments, implements or accessories specially adapted for surgery or diagnosis and not covered by any of the groups , e.g. for luxation treatment or for protecting wound edges
91.
MODULAR ADJUSTABLE BIOREACTOR FOR DECELLULARIZATION AND CELL SEEDING
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Chiang, Tendy
Byun, Woo Yul
Liu, Lumei
Abstract
An adjustable, modular bioreactor has been developed for both partial or full decellularization of organs or tissues and recellularization of the decellularized organs or tissues. The allows for the use of different end pieces to secure different diameters of the tissue to be treated, and is adjustable in length, for example, using a slidable chamber housing, which can be shortened or lengthened merely by sliding of an endpiece, where the chamber remains sealed through the use of an O-ring or other seal and the endpiece is secured in position with a clamp.
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Goodman, Steven D.
Bakaletz, Lauren O.
Partida-Sanchez, Santiago
Abstract
Provided herein is a synthetic polypeptide derived from High Mobility Group Box 1 (HMGB 1) host protein that can both disrupt bacterial biofilms and prevent Neutrophil Extracellular Trap (NET) formation. Also provided herein are methods to disrupt aberrant or excessive NET formation that are particularly well-suited to treat high-risk populations such as those infected with SARS CoV-2, sepsis, autoimmune diseases e.g., systemic lupus erythematosus, rheumatoid arthritis, Type I diabetes mellitus, small vessel vasculitis, autoinflammatory diseases e.g., gout, inflammatory bowel disease, and metabolic diseases e.g., Type 2 diabetes and obesity.
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
A61K 39/40 - AntibodiesImmunoglobulinsImmune serum, e.g. antilymphocytic serum bacterial
A61P 29/00 - Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agentsNon-steroidal antiinflammatory drugs [NSAID]
A61P 31/00 - Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
A61P 37/00 - Drugs for immunological or allergic disorders
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
C12N 15/63 - Introduction of foreign genetic material using vectorsVectorsUse of hosts thereforRegulation of expression
C12N 15/85 - Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
C12P 21/02 - Preparation of peptides or proteins having a known sequence of two or more amino acids, e.g. glutathione
93.
MACROPHAGE POLARIZING ONCOLYTIC HERPES SIMPLEX VIRUS FOR CANCER THERAPY
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Chen, Chun-Yu
Wang, Pin-Yi
Cripe, Timothy P.
Abstract
This disclosure relates to a modified oncolytic herpes simplex virus (oHSV) comprising an expression cassette encoding a histidine-rich glycoprotein (HRG), and uses thereof. One promising avenue for the treatment of cancer is oncolytic virotherapy, e.g., oncolytic Herpes Simplex Virus ( oHSV) which utilizes genetically modified viruses to selectively target and lyse cancer cells while sparing the normal cells. Oncolytic virotherapy is a safe and effective immunotherapeutic platform for different types of cancers.
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Meyer, Kathrin, Christine
Abstract
The disclosure provides gene therapy vectors, such as adeno-associated virus (AAV), designed for treatment of an immunoglobulin-μ binding protein 2 (IGHMBP2)-related disorder.
C12N 15/00 - Mutation or genetic engineeringDNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purificationUse of hosts therefor
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Martin, Paul Taylor
Abstract
The disclosure provides gene therapy vectors, such as adeno-associated virus (AAV), designed for treatment of Lysosomal Acid Lipase Deficiency (LAL-D) disorders such as Wolman Disease and cholesterol ester storage disease (CESD). The disclosed rAAV provide a wild type lipase A (LIRA) cDNA to a subject in need which results in expression of the wild type protein.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
A61K 9/00 - Medicinal preparations characterised by special physical form
A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
A61P 1/16 - Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Lee, Dean Anthony
Kararoudi, Meisam Naeimi
Abstract
Disclosed are self-regulated chimeric antigen receptors and methods of making the same. Also, disclosed herein are methods of treating a cancer through the administration of NK cells and/or NK T cells comprising said chimeric antigen receptors.
C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
C12N 15/85 - Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
97.
COMPOUNDS, COMPOSITIONS, AND METHODS FOR USING HLA-F
THE RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Kaspar, Brian
Abstract
The invention relates to compositions, compounds, methods, and uses for the treatment of amyotrophic lateral sclerosis. In particular, the invention relates to compounds, compositions, methods, and uses for the treatment of amyotrophic lateral sclerosis by increasing the expression of the MHC class I molecule, HLA-F, in motor neurons of the patient.
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
98.
COMPOSITIONS AND METHODS FOR TREATING DISEASE ASSOCIATED WITH DUX4 OVEREXPRESSION
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Saad, Nizar
Harper, Scott Quenton
Abstract
Disclosed herein are products, methods, and uses for treating, ameliorating, delaying the progression of, and/or preventing a muscular dystrophy or a cancer including, but not limited to, facioscapulohumeral muscular dystrophy (FSHD) or a cancer associated with DUX4 expression or overexpression. More particularly, disclosed herein are RNA interference-based products, methods, and uses for inhibiting or downregulating the expression of double homeobox 4 (DUX4). Even more particularly, the disclosure provides microRNA (miRNA) for inhibiting or downregulating the expression of DUX4 and methods of using said miRNA to inhibit or downregulate DUX4 expression in cells and/or in cells of a subject having a muscular dystrophy or a cancer including, but not limited to, FSHD or a cancer associated with DUX4 expression or overexpression. Additionally, the disclosure provides an estrogen, synthetic estrogen, progesterone, progestin, melatonin, bleomycin, pyrazinamide, sorafenib, or a derivative thereof, or a combination of any thereof for upregulating expression of microRNA-675, inhibiting DUX4 expression, and for treating, ameliorating, delaying the progression of, and/or preventing a muscular dystrophy or a cancer including, but not limited to, FSHD or a cancer associated with DUX4 expression or overexpression.
THE RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL (USA)
Inventor
Rajappa, Prajwal
Canella, Alessandro
Abstract
A method of treating or preventing central nervous system cancer in a subject in need thereof is described. The method includes administering to the subject a therapeutically effective amount of myeloid cells modified to express interleukin-2. A population of genetically engineered myeloid cells (GEMys) comprising bone marrow derived myeloid cells that have been genetically modified to express interleukin-2 is also described.
C07H 21/04 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
patents
