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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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Dettagli Bibliografici
Pubblicato in:Hum Gene Ther
Autori principali: 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.
Natura: Artigo
Lingua:Inglês
Pubblicazione: Mary Ann Liebert, Inc. 2018
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Accesso online: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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