Combining Zebrafish and CRISPR/Cas9: Toward a More Efficient Drug Discovery Pipeline.
Combining Zebrafish and CRISPR/Cas9: Toward a More Efficient Drug Discovery Pipeline.
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DOI：
10.3389/fphar.2018.00703
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发表时间：
2018
影响因子：
5.6
通讯作者：
Terriente J
中科院分区：
文献类型：
--
作者：
Cornet C;Di Donato V;Terriente J
在过去的20年里，斑马鱼幼体在基础和应用研究中的应用呈指数级增长。这一成功的原因在于其特定的实验优势：一方面，小尺寸，大量的后代和快速的生命周期极大地促进了大规模的方法，同时保持了3Rs的顺从性;另一方面，与人类的高度遗传和生理同源性以及易于遗传操作使斑马鱼幼虫成为了解人类疾病的高度稳健的模型。总之，这些优势允许使用斑马鱼幼虫进行高通量研究，无论是在化学和遗传表型筛选方面。因此，斑马鱼幼虫作为动物模型被放置在更多的还原论体外高通量筛选和信息丰富，但低通量的临床前试验，使用哺乳动物之间。然而，尽管斑马鱼具有生物学优势和不断增长的翻译验证，但它在目前的药物发现管道中仍然很少使用。在制药行业在将新药推向市场方面面临生产力危机的背景下，斑马鱼和CRISPR/Cas9系统（迄今为止最强大的基因组编辑技术）的综合优势有可能成为简化模拟人类疾病模型生成、验证新药靶点和发现新疗法的宝贵工具。本文将重点介绍CRISPR/Cas9在斑马鱼中的最新进展及其在生物医学研究和药物发现中的所有潜在用途。
The use of zebrafish larvae in basic and applied research has grown exponentially during the last 20 years. The reasons for this success lay in its specific experimental advantages: on the one hand, the small size, the large number of progeny and the fast life cycle greatly facilitate large-scale approaches while maintaining 3Rs amenability; on the other hand, high genetic and physiological homology with humans and ease of genetic manipulation make zebrafish larvae a highly robust model for understanding human disease. Together, these advantages allow using zebrafish larvae for performing high-throughput research, both in terms of chemical and genetic phenotypic screenings. Therefore, the zebrafish larva as an animal model is placed between more reductionist in vitro high-throughput screenings and informative but low-throughput preclinical assays using mammals. However, despite its biological advantages and growing translational validation, zebrafish remains scarcely used in current drug discovery pipelines. In a context in which the pharmaceutical industry is facing a productivity crisis in bringing new drugs to the market, the combined advantages of zebrafish and the CRISPR/Cas9 system, the most powerful technology for genomic editing to date, has the potential to become a valuable tool for streamlining the generation of models mimicking human disease, the validation of novel drug targets and the discovery of new therapeutics. This review will focus on the most recent advances on CRISPR/Cas9 implementation in zebrafish and all their potential uses in biomedical research and drug discovery.
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影响因子：
5.6
作者：
Cornet C;Calzolari S;Miñana-Prieto R;Dyballa S;van Doornmalen E;Rutjes H;Savy T;D'Amico D;Terriente J
通讯作者：
Terriente J
影响因子：
7
作者：
Auer TO;Duroure K;De Cian A;Concordet JP;Del Bene F
通讯作者：
Del Bene F
斑马鱼裂口的模型。
DOI：
10.1002/dvdy.24566
发表时间：
2017-11
期刊：
Developmental dynamics : an official publication of the American Association of Anatomists
影响因子：
--
作者：
Duncan KM;Mukherjee K;Cornell RA;Liao EC
通讯作者：
Liao EC
影响因子：
3.7
作者：
Ali S;van Mil HG;Richardson MK
通讯作者：
Richardson MK
影响因子：
14.9
作者：
Brinkman EK;Chen T;Amendola M;van Steensel B
通讯作者：
van Steensel B