Advances in genome editing for genetic hearing loss.
Advances in genome editing for genetic hearing loss.
复制标题
基因组编辑治疗遗传性听力损失的进展。
DOI：
10.1016/j.addr.2020.05.001
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发表时间：
2021-01
影响因子：
16.1
通讯作者：
Gao X
中科院分区：
文献类型：
--
作者：
Ding N;Lee S;Lieber-Kotz M;Yang J;Gao X
根据世界卫生组织的数据，听力损失影响着全球超过4.66亿人，是最常见的人类感官障碍。据估计，遗传因素导致约50%的先天性听力损失。然而，逆转或预防遗传性听力障碍的治疗方法仍然有限。成簇的规则间隔短回文重复序列相关蛋白9（CRISPR-Cas9）系统能够以高度通用的方式进行可编程和靶向基因编辑，并为遗传性听力损失提供新的基因治疗策略。在本文中，我们总结了最常见的癌症相关基因，说明了使用CRISPR-Cas 9系统进行靶向基因编辑的最新策略，并进一步比较了CRISPR策略与非CRISPR基因疗法。我们还研究了基因组编辑剂的不同载体和递送形式的优点。最后，我们描述了动物模型的发展，这些模型可以促进CRISPR技术在治疗遗传性听力疾病方面的最终临床应用。
According to the World Health Organization, hearing loss affects over 466 million people worldwide and is the most common human sensory impairment. It is estimated that genetic factors contribute to the causation of approximately 50% of congenital hearing loss. Yet, curative approaches to reversing or preventing genetic hearing impairment are still limited. The clustered regularly interspaced short palindromic repeats-associated protein 9 (CRISPR-Cas9) systems enable programmable and targeted gene editing in highly versatile manners and offer new gene therapy strategies for genetic hearing loss. Here, we summarize the most common deafness-associated genes, illustrate recent strategies undertaken by using CRISPR-Cas 9 systems for targeted gene editing and further compare the CRISPR strategies to non-CRISPR gene therapies. We also examine the merits of different vehicles and delivery forms of genome editing agents. Lastly, we describe the development of animal models that could facilitate the eventual clinical applications of the CRISPR technology to the treatment of genetic hearing diseases.
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影响因子：
16.6
作者：
Bowl MR;Simon MM;Ingham NJ;Greenaway S;Santos L;Cater H;Taylor S;Mason J;Kurbatova N;Pearson S;Bower LR;Clary DA;Meziane H;Reilly P;Minowa O;Kelsey L;International Mouse Phenotyping Consortium;Tocchini-Valentini GP;Gao X;Bradley A;Skarnes WC;Moore M;Beaudet AL;Justice MJ;Seavitt J;Dickinson ME;Wurst W;de Angelis MH;Herault Y;Wakana S;Nutter LMJ;Flenniken AM;McKerlie C;Murray SA;Svenson KL;Braun RE;West DB;Lloyd KCK;Adams DJ;White J;Karp N;Flicek P;Smedley D;Meehan TF;Parkinson HE;Teboul LM;Wells S;Steel KP;Mallon AM;Brown SDM
通讯作者：
Brown SDM
影响因子：
64.8
作者：
Anders, Carolin;Niewoehner, Ole;Duerst, Alessia;Jinek, Martin
通讯作者：
Jinek, Martin
影响因子：
11.1
作者：
Al-Moyed, Hanan;Cepeda, Andreia P.;Reisinger, Ellen
通讯作者：
Reisinger, Ellen
DOI：
10.1073/pnas.2334417100
发表时间：
2003-11-25
影响因子：
11.1
作者：
Belyantseva, IA;Boger, ET;Friedman, TB
通讯作者：
Friedman, TB
影响因子：
5.2
作者：
Chakchouk, Imen;Zhang, Di;Leal, Suzanne M.
通讯作者：
Leal, Suzanne M.