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2024
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Invention
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Gene therapy for neurodegenerative disorders.
The disclosure relates, in some aspects, to compos... |
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Invention
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Gene therapies for lysosomal disorders.
The disclosure relates to compositions and methods for t... |
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2023
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Invention
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Gene therapies for lysosomal disorders.
The disclosure relates, in some aspects, to compositions... |
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2022
|
Invention
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Modulation of aav-based gene expression.
Aspects of the disclosure relate to compositions and me... |
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Invention
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Modulation of aav-based gene expression. Aspects of the disclosure relate to compositions and met... |
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2021
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Invention
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Gene therapies for neurodegenerative disease.
The disclosure relates, in some aspects, to compos... |
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Invention
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Gene therapies for neurodegenerative disease. APOE APOE gene or a portion thereof, or any combina... |
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Invention
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Assay for measuring potency of gene therapy drug product.
Disclosed herein is a cell-based assay... |
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Invention
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Assay for measuring potency of gene therapy drug product. Disclosed herein is a cell-based assay ... |
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Invention
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Recombinant adeno-associated virus compositions and methods for producing same.
Disclosed herein... |
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Invention
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Recombinant adeno-associated virus compositions and methods for producing same. Disclosed herein ... |
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Invention
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Gene therapies for neurodegenerative disorders.
The disclosure relates to compositions and metho... |
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Invention
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Gene therapies for lysosomal disorders. The disclosure relates to compositions and methods for tr... |
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Invention
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Gene therapies for neurodegenerative disorders. The disclosure relates to compositions and method... |
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Invention
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Aav vectors encoding parkin and uses thereof.
The disclosure relates, in some aspects, to compos... |
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Invention
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Aav vectors encoding parkin and uses thereof. The disclosure relates, in some aspects, to composi... |
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2020
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Invention
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Gene therapies for lysosomal disorders. The disclosure relates, in some aspects, to compositions ... |
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Invention
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Compositions and methods for treatment of friedreichs ataxia.
The present application provides c... |
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Invention
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Gene therapy for neurodegenerative disorders. This Application is a continuation of international... |
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2019
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Invention
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Gene therapies for neurodegenerative disease. The disclosure relates, in some aspects, to composi... |
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P/S
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Pharmaceutical preparations for the treatment of neurodegenerative diseases and lysosomal disorde... |
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P/S
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Pharmaceutical preparations for the treatment of
neurodegenerative diseases and lysosomal disord... |
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Invention
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Pet imaging ligands for in vivo detection of gba1. e.gin vivoe.ge.g., within the CNS of a subject). |
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2018
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Invention
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Gene therapies for neurodegenerative diseases.
The disclosure relates, in some aspects, to compo... |
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Invention
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Gene therapies for neurodegenerative disease. e.gC9orf72, TMEM106B, ATNX2, RPS25, etc.C9orf72, TM... |
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Invention
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Gene therapies for lysosomal disorders. SCNATMEM106B TMEM106B or a portion thereof, or any combin... |
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Invention
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Modified lysosomal enzymes. Provided herein are lysosomal enzymes comprising modifications that i... |
