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...
Autores principales: | , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Bentham Science Publishers
2019
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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 |