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Therapeutic exon skipping for dysferlinopathies?

Antisense-mediated exon skipping is a promising therapeutic approach for Duchenne muscular dystrophy (DMD) currently tested in clinical trials. The aim is to reframe dystrophin transcripts using antisense oligonucleotides (AONs). These hide an exon from the splicing machinery to induce exon skipping...

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Detalles Bibliográficos
Autores principales:	Aartsma-Rus, Annemieke, Singh, Kavita H K, Fokkema, Ivo F A C, Ginjaar, Ieke B, van Ommen, Gert-Jan, Dunnen, Johan T den, van der Maarel, Silvère M
Formato:	Texto
Lenguaje:	English
Publicado:	Nature Publishing Group 2010
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2987387/ https://www.ncbi.nlm.nih.gov/pubmed/20145676 http://dx.doi.org/10.1038/ejhg.2010.4

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