脊髓性肌萎缩
RNA剪接
形状记忆合金*
选择性拼接
神经科学
计算生物学
外显子
医学
生物
生物信息学
计算机科学
核糖核酸
遗传学
基因
算法
作者
Hasane Ratni,Lutz Mueller,Martin Ebeling
出处
期刊:Progress in Medicinal Chemistry
日期:2019-01-01
卷期号:: 119-156
被引量:7
标识
DOI:10.1016/bs.pmch.2018.12.003
摘要
Targeting RNA drastically expands our target space to therapeutically modulate numerous cellular processes implicated in human diseases. Of particular interest, drugging pre-mRNA splicing appears a very viable strategy; to control levels of splicing product by promoting the inclusion or exclusion of exons. After describing the concept of "splicing modulation", this chapter will cover the outstanding progress achieved in this field, by highlighting the breakthrough accomplished recently for the treatment of spinal muscular atrophy using two therapeutic modalities: splice switching oligonucleotides and small molecules. This review discusses the vital but feasible requirement for such drugs to deliver selectivity, and critical safety aspects are highlighted. Transformational medicines such as those developed to treat SMA are likely just the beginning of this story.
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