Comparison of mutation profiles in the Duchenne muscular dystrophy gene among populations: implications for potential molecular therapies.

doi:10.3390/ijms16035334

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Article

Comparison of mutation profiles in the Duchenne muscular dystrophy gene among populations: implications for potential molecular therapies.

International journal of molecular sciences - 9 Mar 2015

López-Hernández Luz Berenice, Gómez-Díaz Benjamín, Luna-Angulo Alexandra Berenice, Anaya-Segura Mónica, Bunyan David John, Zúñiga-Guzman Carolina, Escobar-Cedillo Rosa Elena, Roque-Ramírez Bladimir, Ruano-Calderón Luis Angel, Rangel-Villalobos Héctor, López-Hernández Julia Angélica, Estrada-Mena Francisco Javier, García Silvia, Coral-Vázquez Ramón Mauricio

Abstract excerpt

Novel therapeutic approaches are emerging to restore dystrophin function in Duchenne Muscular Dystrophy (DMD), a severe neuromuscular disease characterized by progressive muscle wasting and weakness. Some of the molecular therapies, such as exon skipping, stop codon read-through and internal ribosome entry site-mediated translation rely on the type and location of mutations. Hence, their potential applicability...

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PMID: 25761239
DOI: 10.3390/ijms16035334
PMCID: PMC4394478

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