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Teaching an Old Molecule New Tricks: Drug Repositioning for Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is one of the most severe forms of inherited muscular dystrophies. The disease is caused by the lack of dystrophin, a structurally essential protein; hence, a definitive cure would necessarily have to pass through some form of gene and/or cell therapy. Cell- and gen...

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Bibliographic Details
Published in:Int J Mol Sci
Main Authors: Vitiello, Libero, Tibaudo, Lucia, Pegoraro, Elena, Bello, Luca, Canton, Marcella
Format: Artigo
Language:Inglês
Published: MDPI 2019
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Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC6929176/
https://ncbi.nlm.nih.gov/pubmed/31801292
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ijms20236053
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