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Emerging genetic therapies to treat Duchenne muscular dystrophy

PURPOSE OF REVIEW: Duchenne muscular dystrophy is a progressive muscle degenerative disease caused by dystrophin mutations. The purpose of this review is to highlight two emerging therapies designed to repair the primary genetic defect, called `exon skipping' and `nonsense codon suppression...

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Autors principals: Nelson, Stanley F., Crosbie, Rachelle H., Miceli, M. Carrie, Spencer, Melissa J.
Format: Artigo
Idioma:Inglês
Publicat: 2009
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC2856442/
https://ncbi.nlm.nih.gov/pubmed/19745732
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1097/WCO.0b013e32832fd487
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