VWA3A-derived ependyma promoter drives increased therapeutic protein secretion into the CSF

VWA3A-derived ependyma promoter drives increased therapeutic protein secretion into the CSF

Recombinant adeno-associated viral vectors (rAAVs) are a promising strategy to treat neurodegenerative diseases because of their ability to infect non-dividing cells and confer long-term transgene expression. Despite an ever-growing library of capsid variants, widespread delivery of AAVs in the adul...

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Bibliographic Details
Main Authors: Ellie M. Carrell, Yong Hong Chen, Paul T. Ranum, Stephanie L. Coffin, Larry N. Singh, Luis Tecedor, Megan S. Keiser, Eloise Hudry, Bradley T. Hyman, Beverly L. Davidson
Format: Article
Language:English
Published: Elsevier 2023-09-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: Delivery Strategies
promoter
ependyma
AAV
neurodegeneration
central nervous system
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253123001890

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http://www.sciencedirect.com/science/article/pii/S2162253123001890

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