Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN

Neurotropic adeno-associated virus (AAV) serotypes such as AAV9 have been demonstrated to transduce spinal alpha motor neurons when administered intravenously (i.v.) at high doses. This observation led to the recent successful application of i.v. AAV9 delivery to treat infants with spinal muscular a...

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Detalhes bibliográficos
Publicado no:Hum Gene Ther
Main Authors: Hinderer, Christian, Katz, Nathan, Buza, Elizabeth L., Dyer, Cecilia, Goode, Tamara, Bell, Peter, Richman, Laura K., Wilson, James M.
Formato: Artigo
Idioma:Inglês
Publicado em: Mary Ann Liebert, Inc. 2018
Assuntos:
Research Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5865262/
https://ncbi.nlm.nih.gov/pubmed/29378426
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2018.015
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