生物
寡核苷酸
核糖核酸酶
核苷酸
核糖核酸
遗传学
生物化学
DNA
基因
作者
Kenji Rowel Q. Lim,Toshifumi Yokota
出处
期刊:Methods in molecular biology
日期:2020-01-01
卷期号:: 3-19
被引量:15
标识
DOI:10.1007/978-1-0716-0771-8_1
摘要
Gapmers are antisense oligonucleotides composed of a central DNA segment flanked by nucleotides of modified chemistry. Hybridizing with transcripts by sequence complementarity, gapmers recruit ribonuclease H and induce target RNA degradation. Since its concept first emerged in the 1980s, much work has gone into developing gapmers for use in basic research and therapy. These include improvements in gapmer chemistry, delivery, and therapeutic safety. Gapmers have also successfully entered clinical trials for various genetic disorders, with two already approved by the U.S. Food and Drug Administration for the treatment of familial hypercholesterolemia and transthyretin amyloidosis-associated polyneuropathy. Here, we review the events surrounding the early development of gapmers, from conception to their maturity, and briefly conclude with perspectives on their use in therapy.
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