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2025
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Invention
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Treatment of eye diseases and diagnostics. e.g.,OPA1e.g., e.g., low-contrast visual acuity test) ... |
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Invention
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Compounds for treatment of conditions and diseases.
Compounds are provided herein that can promo... |
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2024
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Invention
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Methods for treating conditions and diseases.
Provided herein are methods for treating condition... |
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Invention
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Methods for treating conditions and diseases. SCN1ASCN1A gene and modulating the expression level... |
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Invention
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Opa1 antisense oligomers for treatment of conditions and diseases. Alternative splicing events in... |
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Invention
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Opa1 antisense oligomers for treatment of conditions and diseases.
Agents that target a processe... |
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Invention
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Compounds and methods for treating human subjects. Described herein are compounds and methods tha... |
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Invention
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Antisense oligomer formulations. VVV1.1 protein in the subject. |
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2023
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Invention
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Antisense oligomers for treatment of non-sense mediated rna decay based conditions and diseases. ... |
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Invention
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Antisense oligomers for treatment of conditions and diseases.
Alternative splicing events in SCN... |
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Invention
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Opa1 antisense oligomers for treatment of conditions and diseases.
Alternative splicing events i... |
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Invention
|
Compositions for treatment of conditions and diseases associated with polycystin expression.
Alt... |
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Invention
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Compounds for treatment of conditions and diseases. SCN1ASCN1ASCN1A gene and can modulate the exp... |
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2022
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Invention
|
Opa1 antisense oligomers for treatment of conditions and diseases. e.g.e.g.e.g., via modulation o... |
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Invention
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Compositions and methods for modulating splicing and protein expression.
Alternative splicing ev... |
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Invention
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Compositions for treatment of conditions and diseases associated with polycystin expression. Alte... |
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2021
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Invention
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Methods and compositions for modulating splicing of alternative introns.
Provided herein are met... |
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Invention
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Methods and compositions for modulating splicing and translation.
Alternative splicing events in... |
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Invention
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Antisense oligomers and uses thereof.
Provided herein are methods and compositions for increasin... |
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Invention
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Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome.... |
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2020
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Invention
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Antisense oligomers for treatment of conditions and diseases. Alternative splicing events in SCN1... |
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Invention
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Antisense oligomers for treatment of conditions and diseases. SCN1ASCN1ASCN1A gene can modulate t... |
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Invention
|
Compositions and methods for modulating splicing and protein expression. Alternative splicing eve... |
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Invention
|
Methods and compositions for modulating splicing of alternative introns. Provided herein are meth... |
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Invention
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Methods and compositions for modulating splicing and translation. Alternative splicing events in ... |
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2019
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Invention
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Methods and compositions for treatment of cholesteryl ester storage disease. Provided herein are ... |
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2018
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Invention
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Antisense oligomers and uses thereof. Provided herein are methods and compositions for increasing... |
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2016
|
Invention
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Compositions and methods for treatment of liver diseases. Provided herein are methods and composi... |
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Invention
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Compositions and methods for treatment of kidney diseases. Provided herein are methods and compos... |
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Invention
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Compositions and methods for treatment of retinitis pigmentosa 18 and retinitis pigmentosa 13. Pr... |
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Invention
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Antisense oligomers for treatment of alagille syndrome. Provided herein are methods and compositi... |
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Invention
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Antisense oligomers for treatment of polycystic kidney disease. Provided herein are methods and c... |
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G/S
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Pharmaceuticals and medical preparations. Scientific research and development; Research and devel... |
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G/S
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Pharmaceutical preparations for the treatment of inherited diseases and genetic diseases, namely ... |
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G/S
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Research and development in the field of bioscience |
