Therapeutic potential of antisense oligonucleotides as modulators of alternative splicing

被引：183

作者：

Sazani, P

Kole, R

机构：

[1] Univ N Carolina, Lineberger Comprehens Canc Ctr, Chapel Hill, NC 27599 USA

[2] Univ N Carolina, Dept Pharmacol, Chapel Hill, NC 27599 USA

来源：

JOURNAL OF CLINICAL INVESTIGATION | 2003年 / 112卷 / 04期

关键词：

D O I：

10.1172/JCI200319547

中图分类号：

R-3 [医学研究方法]; R3 [基础医学];

学科分类号：

1001 ;

摘要：

An estimated 60% of all human genes undergo alternative splicing, a highly regulated process that pro, duces splice variants with different functions. Such variants have been linked to a variety of cancers, and genetic diseases such as thalassemia and cystic fibrosis. This Perspective describes a promising approach to RNA repair based on the use of antisense oligonucleotides to modulate alternative splicing and engender the production of therapeutic gene products.

引用

页码：481 / 486

页数：6

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