Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector

Effective gene delivery to the central nervous system (CNS) is vital for development of novel gene therapies for neurological diseases. Adeno-associated virus (AAV) vectors have emerged as an effective platform for in vivo gene transfer, but overall neuronal transduction efficiency of vectors derive...

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Pubblicato in:Mol Ther
Autori principali: Choudhury, Sourav R, Harris, Anne F, Cabral, Damien J, Keeler, Allison M, Sapp, Ellen, Ferreira, Jennifer S, Gray-Edwards, Heather L, Johnson, Jacob A, Johnson, Aime K, Su, Qin, Stoica, Lorelei, DiFiglia, Marian, Aronin, Neil, Martin, Douglas R, Gao, Guangping, Sena-Esteves, Miguel
Natura: Artigo
Lingua:Inglês
Pubblicazione: Nature Publishing Group 2016
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Original Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC4886933/
https://ncbi.nlm.nih.gov/pubmed/26708003
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2015.231
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