期刊
CELL
卷 148, 期 6, 页码 1085-1088出版社
CELL PRESS
DOI: 10.1016/j.cell.2012.02.014
关键词
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资金
- Duchenne Parent Project (the Netherlands)
- ZonMw (the Netherlands)
- Prinses Beatrix Foundation
- FP7 BIO-NMD project [HEALTH-F2-2009-241665]
Dysregulation of splicing and alternative splicing underlies many genetic and acquired diseases. We present an overview of recent strategies and successes in modulating splicing therapeutically in clinical and preclinical contexts. Effective approaches include restoring open reading frames, influencing alternative splicing, and inducing exon inclusion to generate beneficial proteins and remove deleterious ones.
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