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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...
Autores principales: | , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Cold Spring Harbor Laboratory
2023
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Materias: | |
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 |