In vivo selection in non-human primates identifies superior AAV capsids for on-target CSF delivery to spinal cord

Systemic administration of adeno-associated virus (AAV) vectors for spinal cord gene therapy has challenges including toxicity at high doses and pre-existing immunity that reduces efficacy. Intrathecal delivery of AAV vectors into the cerebral spinal fluid (CSF) can avoid many of the issues of syste...

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Detalles Bibliográficos
Autores principales: Hanlon, Killian S., Cheng, Ming, De La Cruz, Demitri, Patel, Nikita, Santoscoy, Miguel C., Gong, Yi, Ng, Carrie, Nguyen, Diane M., Nammour, Josette, Clark, Sean W., Kozarsky, Karen, Maguire, Casey A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Cold Spring Harbor Laboratory 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10515928/
https://www.ncbi.nlm.nih.gov/pubmed/37745398
http://dx.doi.org/10.1101/2023.09.13.557506

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