Innovative Therapeutic Strategies for Cystic Fibrosis: Moving Forward to CRISPR Technique.
Innovative Therapeutic Strategies for Cystic Fibrosis: Moving Forward to CRISPR Technique.
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DOI：
10.3389/fphar.2018.00396
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发表时间：
2018
影响因子：
5.6
通讯作者：
Pistritto G
中科院分区：
文献类型：
--
作者：
Marangi M;Pistritto G
CRISPR-CAS9是近年来分子生物学领域最具革命性的技术之一。CRISPR技术是一种很有前途的基因编辑工具，它为研究人员提供了轻松改变DNA序列和修改基因功能的机会。它的许多潜在应用包括纠正基因缺陷，治疗和防止疾病的传播。囊性纤维化是由囊性纤维化跨膜电导调节基因突变引起的最常见的致死性遗传病之一。虽然CF是老相识，但目前仍没有有效/彻底的治疗方法。由于各种治疗方法的结合，预期寿命有所提高，但总体上低于平均水平。最近，许多额外的关键药物已经在欧洲获得了治疗CF的许可，其中包括CFTR调节剂。但是，创新的基因指导治疗已经开始，可以预见的是，这将在未来几十年内迅速改善CF的临床疾病和存活率。因此，CRISPR-Cas9方法可能是修复CFTR突变的一种有效工具，并在组织和动物CF病模型中获得了可望的结果。
One of the most revolutionary technologies in recent years in the field of molecular biology is CRISPR-Cas9. CRISPR technology is a promising tool for gene editing that provides researchers the opportunity to easily alter DNA sequences and modify gene function. Its many potential applications include correcting genetic defects, treating and preventing the spread of diseases. Cystic fibrosis (CF) is one of the most common lethal genetic diseases caused by mutations in the CF transmembrane conductance regulator (CFTR) gene. Although CF is an old acquaintance, there is still no effective/resolutive cure. Life expectancy has improved thanks to the combination of various treatments, but it is generally below average. Recently, a significant number of additional key medications have become licensed in Europe for the CF treatment including CFTR modulators. But innovative genomically-guided therapies have begun for CF and it is predictable that this will lead to rapid improvements in CF clinical disease and survival in the next decades. In this way, CRISPR-Cas9 approach may represent a valid tool to repair the CFTR mutation and hopeful results were obtained in tissue and animal models of CF disease.
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影响因子：
8.8
作者：
Firth AL;Menon T;Parker GS;Qualls SJ;Lewis BM;Ke E;Dargitz CT;Wright R;Khanna A;Gage FH;Verma IM
通讯作者：
Verma IM
DOI：
10.1126/science.aad5143
发表时间：
2016-01-22
期刊：
Science (New York, N.Y.)
影响因子：
--
作者：
Nelson CE;Hakim CH;Ousterout DG;Thakore PI;Moreb EA;Castellanos Rivera RM;Madhavan S;Pan X;Ran FA;Yan WX;Asokan A;Zhang F;Duan D;Gersbach CA
通讯作者：
Gersbach CA
CRISPR/Cas9介导的人类三核受精卵基因编辑
影响因子：
21.1
作者：
Liang, Puping;Xu, Yanwen;Zhang, Xiya;Ding, Chenhui;Huang, Rui;Zhang, Zhen;Lv, Jie;Xie, Xiaowei;Chen, Yuxi;Li, Yujing;Sun, Ying;Bai, Yaofu;Songyang, Zhou;Ma, Wenbin;Zhou, Canquan;Huang, Junjiu
通讯作者：
Huang, Junjiu
影响因子：
5.1
作者：
Griesenbach, U;Geddes, DM;Alton, EWFW
通讯作者：
Alton, EWFW
影响因子：
4.4
作者：
Bulloch, Marilyn N.;Hanna, Cameron;Giovane, Richard
通讯作者：
Giovane, Richard