Evaluation of Intrathecal Routes of Administration for Adeno-Associated Viral Vectors in Large Animals

Delivery of adeno-associated viral (AAV) vectors into the cerebrospinal fluid (CSF) can achieve gene transfer to cells throughout the brain and spinal cord, potentially making many neurological diseases tractable gene therapy targets. Identifying the optimal route of CSF access for intrathecal AAV d...

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Udgivet i:Hum Gene Ther
Main Authors: Hinderer, Christian, Bell, Peter, Katz, Nathan, Vite, Charles H., Louboutin, Jean-Pierre, Bote, Erin, Yu, Hongwei, Zhu, Yanqing, Casal, Margret L., Bagel, Jessica, O'Donnell, Patricia, Wang, Ping, Haskins, Mark E., Goode, Tamara, Wilson, James M.
Format: Artigo
Sprog:Inglês
Udgivet: Mary Ann Liebert, Inc. 2018
Fag:
Research Articles
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5770082/
https://ncbi.nlm.nih.gov/pubmed/28806897
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2017.026
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