Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model

Duchenne muscular dystrophy is an X-linked muscle disease characterized by mutations in the dystrophin gene. Many of these can be corrected at the posttranscriptional level by skipping the mutated exon. We have obtained persistent exon skipping in mdx mice by tail vein injection with an adeno-associ...

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Detalhes bibliográficos
Main Authors: Denti, Michela Alessandra, Rosa, Alessandro, D’Antona, Giuseppe, Sthandier, Olga, De Angelis, Fernanda Gabriella, Nicoletti, Carmine, Allocca, Mariacarmela, Pansarasa, Orietta, Parente, Valeria, Musarò, Antonio, Auricchio, Alberto, Bottinelli, Roberto, Bozzoni, Irene
Formato: Artigo
Idioma:Inglês
Publicado em: National Academy of Sciences 2006
Assuntos:
Biological Sciences
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC1450150/
https://ncbi.nlm.nih.gov/pubmed/16501048
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.0508917103
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