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2025
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Invention
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Acid-alpha glucosidase variants and uses thereof.
The present invention relates to variants of a... |
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2024
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Invention
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Hybrid regulatory elements.
The present invention relates to hybrid transcription regulatory ele... |
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Invention
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Methods for aav vector re-administration. The invention relates to a combination of a recombinant... |
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Invention
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Prediction of multiple aav properties by deep learning. The invention relates to a method impleme... |
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Invention
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Novel optimized utrophin micro-genes. The present invention concerns a micro-utrophin comprising ... |
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Invention
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Novel mididystrophins. The present invention concerns mididystrophins comprising at least the rod... |
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Invention
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N-terminal truncated glycogen debranching enzymes for the treatment of glycogen storage disease i... |
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Invention
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Hybrid recombinant adeno-associated virus serotype between aav9 and aavrh74 with reduced liver tr... |
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Invention
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Methods and tools for detecting the impact of compounds on protein, rna or dna interactions. in v... |
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Invention
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Microfluidic device for use in a system for measuring at least one mechanical property of at leas... |
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Invention
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Induction of immune tolerance by aav vector comprising the combination of a liver detargeted caps... |
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Invention
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Method for detecting peptide biomarkers. The invention relates in particular to a method for dete... |
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2023
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Invention
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Methods for the production of human melanocytes from pluripotent stem cells. The present inventio... |
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Invention
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Lsd1 inhibitor and prmt6 inhibitor for use in the treatment of a disease associated with gain-of-... |
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Invention
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Closed device for culturing and transporting cell therapy product. The present invention relates ... |
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Invention
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Hybrid aav vector enhancing transgene expression in the liver. The invention relates to a hybrid ... |
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Invention
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Immunotherapy of skeletal myopathies using anti-fap car-t cells. The invention relates to an immu... |
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Invention
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N-terminal truncated gde for the treatment of glycogen storage disease iii. The present invention... |
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Invention
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Peptide-modified aav capsid with enhanced muscle transduction efficiency. The invention relates t... |
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Invention
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Use of bazedoxifene for increasing muscle survival. The invention relates to the use of bazedoxif... |
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Invention
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Novel polynucleotides encoding a human fkrp protein.
The present invention concerns synthetic po... |
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2022
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Invention
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Haloperidol for use in the treatment of spinal muscular atrophy.
Haloperidol, or a pharmaceutica... |
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Invention
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Haloperidol for use in the treatment of spinal muscular atrophy. Haloperidol, or a pharmaceutical... |
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Invention
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Gamma-sarcoglycan gene transfer increase using modified itr sequences. The present invention rela... |
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Invention
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Calpain-3 gene transfer increase using modified itr sequences. The present invention relates to a... |
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Invention
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Cylinder with integral position sensor.
A cylinder actuator includes two elements mobile one rel... |
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Invention
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Treatment of glycogen storage disease iii.
The present invention relates to vectors and composit... |
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Invention
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Stable pseudotyped lentiviral particles and uses thereof.
The present invention relates to a met... |
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Invention
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Microstructured scaffold for the preparation of a tissue implant.
The preparation of a microstru... |
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Invention
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Automatic method for segmentation of a thrombus and a lesion in a three-dimensional brain image. ... |
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Invention
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Use of bazedoxifene for increasing muscle survival.
The invention concerns the use of bazedoxife... |
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2021
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Invention
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Lysosomal acid lipase variants and uses thereof.
The present invention relates to variants of ly... |
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Invention
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Enhancing utrophin expression in cell by inducing mutations within utrophin regulatory elements a... |
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Invention
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Bifunctional lentiviral vectors allowing the bs-globin silencing and expression of an anti-sickli... |
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Invention
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Peptide-modified aav capsid.
The invention relates to a peptide-modified AAV capsid with improve... |
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Invention
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C-terminal truncated gde for the treatment of glycogen storage disease iii.
The present inventio... |
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Invention
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Synthesis of composite beads coated with layered manganese oxide and use of such beads to remove ... |
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Invention
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A novel muscle-specific promoter.
The present invention concerns a novel short promoter characte... |
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Invention
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Method for engineering novel hybrid aav capsids through hyper variable regions swapping.
The inv... |
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Invention
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Gene therapy expression system allowing an adequate expression in the muscles and in the heart of... |
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Invention
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Treatment of dilated cardiomyopathies.
The invention relates to the treatment of dilated cardiom... |
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Invention
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Treatment of genetic dilated cardiomyopathies.
The invention relates to the treatment of genetic... |
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Invention
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Nrf2 activator for use in treating dilated cardiomyopathies.
The present invention relates to th... |
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Invention
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Cilp-1 inhibitors for use in the treatment of dilated cardiomyopathies.
The present disclosure r... |
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Invention
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Vectors encoding a glucose-6-phosphatase (g6pase-a) for gene therapy.
The invention relates to a... |
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Invention
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Methods for preventing induction of immune responses to the transduced cells expressing a transge... |
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2020
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Invention
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Combination of a proteasome inhibitor and a hdac inhibitor and its use for the treatment of genet... |
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Invention
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Efficient and rapid specification of spinal neuronal subtypes from human pluripotent stem cells. ... |
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Invention
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Gene therapy expression system alleviating cardiac toxicity of fkrp.
The present invention conce... |
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Invention
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Gene therapy of fibroblast growth factor 23 related hypophosphatemic diseases.
The invention rel... |
