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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Detalles Bibliográficos
Autores principales: Carrell, Ellie M., Chen, Yong Hong, Ranum, Paul T., Coffin, Stephanie L., Singh, Larry N., Tecedor, Luis, Keiser, Megan S., Hudry, Eloise, Hyman, Bradley T., Davidson, Beverly L.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10400871/
https://www.ncbi.nlm.nih.gov/pubmed/37547292
http://dx.doi.org/10.1016/j.omtn.2023.07.016

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10400871/
https://www.ncbi.nlm.nih.gov/pubmed/37547292
http://dx.doi.org/10.1016/j.omtn.2023.07.016

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