反义寡核苷酸作为可变剪接调节剂的治疗潜力。
Therapeutic potential of antisense oligonucleotides as modulators of alternative splicing.
作者信息
Sazani Peter, Kole Ryszard
机构信息
University of North Carolina, Lineberger Comprehensive Cancer Center, CB #7295, Chapel Hill, North Carolina 27599-7295, USA.
出版信息
J Clin Invest. 2003 Aug;112(4):481-6. doi: 10.1172/JCI19547.
Abstract
An estimated 60% of all human genes undergo alternative splicing, a highly regulated process that produces 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.
摘要
据估计，所有人类基因中有60%会发生可变剪接，这是一个受到高度调控的过程，会产生具有不同功能的剪接变体。这些变体与多种癌症以及地中海贫血和囊性纤维化等遗传疾病有关。本观点文章描述了一种基于使用反义寡核苷酸来调节可变剪接并产生治疗性基因产物的有前景的RNA修复方法。
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