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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Bibliografiska uppgifter
I publikationen:Hum Gene Ther
Huvudupphovsmän: Hinderer, Christian, Katz, Nathan, Buza, Elizabeth L., Dyer, Cecilia, Goode, Tamara, Bell, Peter, Richman, Laura K., Wilson, James M.
Materialtyp: Artigo
Språk:Inglês
Publicerad: Mary Ann Liebert, Inc. 2018
Ämnen:
Research Articles
Länkar: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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