Systemic Gene Delivery in Large Species for Targeting Spinal Cord, Brain, and Peripheral Tissues for Pediatric Disorders

Adeno-associated virus type 9 (AAV9) is a powerful tool for delivering genes throughout the central nervous system (CNS) following intravenous injection. Preclinical results in pediatric models of spinal muscular atrophy (SMA) and lysosomal storage disorders provide a compelling case for advancing A...

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Detalhes bibliográficos
Main Authors: Bevan, Adam K, Duque, Sandra, Foust, Kevin D, Morales, Pablo R, Braun, Lyndsey, Schmelzer, Leah, Chan, Curtis M, McCrate, Mary, Chicoine, Louis G, Coley, Brian D, Porensky, Paul N, Kolb, Stephen J, Mendell, Jerry R, Burghes, Arthur HM, Kaspar, Brian K
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2011
Assuntos:
Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3222525/
https://ncbi.nlm.nih.gov/pubmed/21811247
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2011.157
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