Splice Modulating Therapies for Human Disease

被引：105

作者：

Spitali, Pietro ^{[1
]}

Aartsma-Rus, Annemieke ^{[1
]}

机构：

[1] Leiden Univ, Med Ctr, Dept Human Genet, NL-2300 RC Leiden, Netherlands

来源：

CELL | 2012年 / 148卷 / 06期

关键词：

DYSTROPHIN RESTORATION; RNA; REPEAT; REVERSAL; RESCUE; BRAIN;

D O I：

10.1016/j.cell.2012.02.014

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

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.

引用

页码：1085 / 1088

页数：4

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