Potential therapeutic applications of antisense morpholino oligonucleotides in modulation of splicing in primary immunodeficiency diseases.
Potential therapeutic applications of antisense morpholino oligonucleotides in modulation of splicing in primary immunodeficiency diseases.
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DOI：
10.1016/j.jim.2010.12.001
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发表时间：
2011-02-28
影响因子：
2.2
通讯作者：
Gatti, Richard A.
中科院分区：
文献类型：
--
作者：
Du, Liutao;Gatti, Richard A.
关键词：
高度互补的反义吗啉代寡核苷酸（AMOs）可以与前体mRNA结合并调节剪接位点选择。这为剪接过程的调控提供了一个强有力的工具，如剪接突变和无义突变的亚型的校正以及选择性剪接过程的重编程。因此，AMO介导的剪接调节代表了遗传性疾病的有吸引力的治疗策略。原发性免疫缺陷病（PID）是一组异质性遗传疾病，由参与免疫系统发育和维持的基因突变引起。这些突变中有许多是剪接突变和无义突变，可以通过AMOs操纵。本文综述了AMO-mediated剪接调控方法及其在PID中的潜在应用。
Highly complementary antisense morpholino oligonucleotides (AMOs) can bind to pre-mRNA and modulate splicing site selection. This offers a powerful tool to regulate splicing process, such as correcting subtypes of splicing mutations and nonsense mutations and reprogramming alternative splicing processes. Therefore, AMO-mediated splicing modulation represents an attractive therapeutic strategy for genetic disorders. Primary immunodeficiency diseases (PIDs) are a heterogeneous group of genetic disorders that result from mutations in genes involved in development and maintenance of the immune system. Many of these mutations are splicing mutations and nonsense mutations that can be manipulated by AMOs. This review discusses AMO-mediated splicing modulation approaches and their potential applications in PIDs.
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DOI：
10.1016/j.bbrc.2009.11.141
发表时间：
2010-01-01
影响因子：
3.1
作者：
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