Therapeutic Modulation of RNA Splicing in Malignant and Non-Malignant Disease

被引：33
作者：
El Marabti, Ettaib [1 ]
Abdel-Wahab, Omar [2 ,3 ]
机构：
[1] Fred Hutchinson Canc Res Ctr, Clin Transplant Res Div, Seattle, WA 98109 USA
[2] Mem Sloan Kettering Canc Ctr, Human Oncol & Pathogenesis Program, New York, NY 10065 USA
[3] Mem Sloan Kettering Canc Ctr, Dept Med, Leukemia Serv, New York, NY 10065 USA
来源：
TRENDS IN MOLECULAR MEDICINE | 2021年 / 27卷 / 07期
基金：
美国国家卫生研究院;
关键词：
PRE-MESSENGER-RNA; SPINAL MUSCULAR-ATROPHY; SURVIVAL MOTOR-NEURON; RESTORES DYSTROPHIN EXPRESSION; ANTISENSE OLIGONUCLEOTIDES; SMN2; GENE; DEVELOPMENTAL DISORDER; MINOR SPLICEOSOME; INTRON MUTATION; SR PROTEIN;
D O I：
10.1016/j.molmed.2021.04.005
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：
RNA splicing is the enzymatic process by which non-protein coding sequences are removed from RNA to produce mature protein-coding mRNA. Splicing is thereby a major mediator of proteome diversity as well as a dynamic regulator of gene expression. Genetic alterations disrupting splicing of individual genes or altering the function of splicing factors contribute to a wide range of human genetic diseases as well as cancer. These observations have resulted in the development of therapies based on oligonucleotides that bind to RNA sequences and modulate splicing for therapeutic benefit. In parallel, small molecules that bind to splicing factors to alter their function or modify RNA processing of individual transcripts are being pursued for monogenic disorders as well as for cancer.
引用
收藏
页码：643 / 659
页数：17
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