Developing AAV-delivered nonsense suppressor tRNAs for neurological disorders

Adeno-associated virus (AAV)-based gene therapy is a clinical stage therapeutic modality for neurological disorders. A common genetic defect in myriad monogenic neurological disorders is nonsense mutations that account for about 11% of all human pathogenic mutations. Stop codon readthrough by suppre...

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Bibliographic Details
Main Authors: Jiaming Wang, Guangping Gao, Dan Wang
Format: Article
Language:English
Published: Elsevier 2024-07-01
Series:Neurotherapeutics
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Online Access:http://www.sciencedirect.com/science/article/pii/S1878747924000771
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