Therapeutic Modulation of RNA Splicing in Malignant and Non-Malignant Disease.
Therapeutic Modulation of RNA Splicing in Malignant and Non-Malignant Disease.
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DOI：
10.1016/j.molmed.2021.04.005
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发表时间：
2021-07
影响因子：
13.6
通讯作者：
Abdel-Wahab O
中科院分区：
文献类型：
--
作者：
El Marabti E;Abdel-Wahab O
RNA剪接是从RNA中去除非蛋白质编码序列以产生成熟的蛋白质编码信使RNA(MRNA)的酶过程。因此，剪接是蛋白质组多样性的主要介体，也是基因表达的动态调节因子。破坏单个基因剪接或改变剪接因子功能的基因改变会导致广泛的人类遗传病和癌症。这些观察结果导致了寡核苷酸疗法的发展，这种疗法与RNA序列结合，以调节剪接以达到治疗效果。同时，与剪接因子结合以改变其功能或改变单个转录本的RNA加工的小分子正在被用于治疗单基因疾病和癌症。
RNA splicing is the enzymatic process by which non-protein coding sequences are removed from RNA to produce mature protein coding messenger RNA (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 oligonucleotide therapies which bind to RNA sequences to modulate splicing for therapeutic benefit. In parallel, small molecules which bind to splicing factors to alter their function or modify RNA processing of individual transcripts are being pursued for monogenic disorders as well as cancer.
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