Homologous Recombination Mediates Functional Recovery of Dysferlin Deficiency following AAV5 Gene Transfer

The dysferlinopathies comprise a group of untreatable muscle disorders including limb girdle muscular dystrophy type 2B, Miyoshi myopathy, distal anterior compartment syndrome, and rigid spine syndrome. As with other forms of muscular dystrophy, adeno-associated virus (AAV) gene transfer is a partic...

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Detalles Bibliográficos
Autores principales: Grose, William E., Clark, K. Reed, Griffin, Danielle, Malik, Vinod, Shontz, Kimberly M., Montgomery, Chrystal L., Lewis, Sarah, Brown, Robert H., Janssen, Paul M. L., Mendell, Jerry R., Rodino-Klapac, Louise R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2012
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3376115/
https://www.ncbi.nlm.nih.gov/pubmed/22720081
http://dx.doi.org/10.1371/journal.pone.0039233

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