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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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
Format: Artigo
Jezik:Inglês
Izdano: National Academy of Sciences 2006
Teme:
Biological Sciences
Online dostop: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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