An Overview of Recent Advances and Clinical Applications of Exon Skipping and Splice Modulation for Muscular Dystrophy and Various Genetic Diseases.
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DOI: 10.1007/978-1-4939-8651-4_2
Abstract: Exon skipping is a therapeutic approach that is feasible for various genetic diseases and has been studied and developed for over two decades. This approach uses antisense oligonucleotides (AON) to modify the splicing of pre-mRNA… read more here.
Keywords: muscular dystrophy; dystrophy; various genetic; genetic diseases ... See more keywords