Combined Treatment with Peptide-Conjugated Phosphorodiamidate Morpholino Oligomer-PPMO and AAV-U7 Rescues the Severe DMD Phenotype in Mice

Duchenne muscular dystrophy (DMD) is a devastating neuromuscular disease caused by an absence of the dystrophin protein, which is essential for muscle fiber integrity. Among the developed therapeutic strategies for DMD, the exon-skipping approach corrects the frameshift and partially restores dystro...

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Dettagli Bibliografici
Pubblicato in:Mol Ther Methods Clin Dev
Autori principali: Forand, Anne, Muchir, Antoine, Mougenot, Nathalie, Sevoz-Couche, Caroline, Peccate, Cécile, Lemaitre, Mégane, Izabelle, Charlotte, Wood, Matthew, Lorain, Stéphanie, Piétri-Rouxel, France
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society of Gene & Cell Therapy 2020
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Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC7177166/
https://ncbi.nlm.nih.gov/pubmed/32346547
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.03.011
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