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: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2011
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3222525/
https://www.ncbi.nlm.nih.gov/pubmed/21811247
http://dx.doi.org/10.1038/mt.2011.157

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3222525/
https://www.ncbi.nlm.nih.gov/pubmed/21811247
http://dx.doi.org/10.1038/mt.2011.157

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