Intramuscular scAAV9-SMN Injection Mediates Widespread Gene Delivery to the Spinal Cord and Decreases Disease Severity in SMA Mice

We have recently demonstrated the remarkable efficiency of self-complementary (sc) AAV9 vectors for central nervous system (CNS) gene transfer following intravenous delivery in mice and larger animals. Here, we investigated whether gene delivery to motor neurons (MNs) could also be achieved via intr...

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Bibliografski detalji
Glavni autori: Benkhelifa-Ziyyat, Sofia, Besse, Aurore, Roda, Marianne, Duque, Sandra, Astord, Stéphanie, Carcenac, Romain, Marais, Thibaut, Barkats, Martine
Format: Artigo
Jezik:Inglês
Izdano: Nature Publishing Group 2013
Teme:
Original Article
Online pristup:https://ncbi.nlm.nih.gov/pmc/articles/PMC3594018/
https://ncbi.nlm.nih.gov/pubmed/23295949
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2012.261
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