Elusive sources of variability of dystrophin rescue by exon skipping

BACKGROUND: Systemic delivery of anti-sense oligonucleotides to Duchenne muscular dystrophy (DMD) patients to induce de novo dystrophin protein expression in muscle (exon skipping) is a promising therapy. Treatment with Phosphorodiamidate morpholino oligomers (PMO) lead to shorter de novo dystrophin...

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Pubblicato in:Skelet Muscle
Autori principali: Vila, Maria Candida, Klimek, Margaret Benny, Novak, James S., Rayavarapu, Sree, Uaesoontrachoon, Kitipong, Boehler, Jessica F., Fiorillo, Alyson A., Hogarth, Marshall W., Zhang, Aiping, Shaughnessy, Conner, Gordish-Dressman, Heather, Burki, Umar, Straub, Volker, Lu, Qi Long, Partridge, Terence A., Brown, Kristy J., Hathout, Yetrib, van den Anker, John, Hoffman, Eric P., Nagaraju, Kanneboyina
Natura: Artigo
Lingua:Inglês
Pubblicazione: BioMed Central 2015
Soggetti:
Research
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC4667482/
https://ncbi.nlm.nih.gov/pubmed/26634117
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13395-015-0070-6
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