Chimeric snRNA molecules carrying antisense sequences against the splice junctions of exon 51 of the dystrophin pre-mRNA induce exon skipping and restoration of a dystrophin synthesis in Δ48-50 DMD cells

Deletions and point mutations in the dystrophin gene cause either the severe progressive myopathy Duchenne muscular dystrophy (DMD) or the milder Becker muscular dystrophy, depending on whether the translational reading frame is lost or maintained. Because internal in-frame deletions in the protein...

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Detalhes bibliográficos
Main Authors: De Angelis, Fernanda Gabriella, Sthandier, Olga, Berarducci, Barbara, Toso, Silvia, Galluzzi, Giuliana, Ricci, Enzo, Cossu, Giulio, Bozzoni, Irene
Formato: Artigo
Idioma:Inglês
Publicado em: National Academy of Sciences 2002
Assuntos:
Biological Sciences
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC123162/
https://ncbi.nlm.nih.gov/pubmed/12077324
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.142302299
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