RNAi Therapy for Dominant Muscular Dystrophies and Other Myopathies
作者： Lindsay M. Wallace , Sara E. Garwick , Scott Q. Harper
DOI: 10.1007/978-1-4419-1207-7_7
关键词:
摘要: Historically, the muscle gene therapy field has been primarily focused on replacing defective or missing genes underlying recessive disorders and matured to point where several replacement strategies have now tested are underway in human clinical trials. Unfortunately, not indicated for treating dominant diseases, reduction elimination of an abnormal allele would be needed, as a result, therapies muscular dystrophies lagged behind. Importantly, emergence RNA interference (RNAi) gene-silencing tool provided means begin closing this development gap. In first edition chapter Muscle Gene Therapy, we discussed prospects combining RNAi treat but proof concept its practical usage had demonstrated at time. Here, second edition, update our current understanding mechanisms RNAi, compile preclinical examples RNAi-based discuss translating these toward clinic.
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