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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Detalles Bibliográficos
Autores principales: 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
Lenguaje:Inglês
Publicado: Nature Publishing Group 2011
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Original Article
Acceso en línea: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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