Extensive and prolonged restoration of dystrophin expression with vivo-morpholino-mediated multiple exon skipping in dystrophic dogs.

Extensive and prolonged restoration of dystrophin expression with vivo-morpholino-mediated multiple exon skipping in dystrophic dogs.

Duchenne muscular dystrophy (DMD) is a severe and the most prevalent form of muscular dystrophy, characterized by rapid progression of muscle degeneration. Antisense-mediated exon skipping is currently one of the most promising therapeutic options for DMD. However, unmodified antisense oligos such a...

Bibliografiset tiedot
Päätekijät:	Yokota, T, Nakamura, A, Nagata, T, Saito, T, Kobayashi, M, Aoki, Y, Echigoya, Y, Partridge, T, Hoffman, E, Takeda, S
Aineistotyyppi:	Journal article
Kieli:	English
Julkaistu:	2012

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