Repurposing Pathogenic Variants of DMD Gene and its Isoforms for DMD Exon Skipping Intervention

BACKGROUND: Duchenne Muscular Dystrophy (DMD) is a progressive, fatal neuromuscular disorder caused by mutations in the DMD gene. Emerging antisense oligomer based exon skipping therapy provides hope for the restoration of the reading frame. OBJECTIVES: Population-based DMD mutation database may ena...

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Detalles Bibliográficos
Autores principales: Tyagi, Rahul, Kumar, Sumit, Dalal, Ashwin, Mohammed, Faruq, Mohanty, Manju, Kaur, Paramvir, Anand, Akshay
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Bentham Science Publishers 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7327972/
https://www.ncbi.nlm.nih.gov/pubmed/32655290
http://dx.doi.org/10.2174/1389202920666191107142754