Splice Modulating Therapies for Human Disease

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• 作者：Pietro Spitali¹ ; Annemieke Aartsma-Rus¹ ; ^{a.m.rus@lumc.nl}
• 刊名：Cell
• 出版年：2012
• 出版时间：16 March, 2012
• 年：2012
• 卷：148
• 期：6
• 页码：1085-1088
• 全文大小：329 K

文摘

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.