清脆的
药物发现
斑马鱼
基因组编辑
计算生物学
转录激活物样效应核酸酶
疾病
Cas9
生物
模式生物
生物信息学
计算机科学
医学
遗传学
基因
病理
作者
Mirjam Keßler,Wolfgang Rottbauer,Steffen Just
标识
DOI:10.1517/17460441.2015.1078788
摘要
Novel genome-editing techniques such as the clustered regularly interspaced short palindromic repeat/Cas9 (CRISPR/Cas9) and transcription activator-like effector nuclease (TALEN) technologies allow highly efficient and reliable disease modeling in the in vivo system zebrafish. The ambition of developing personalized therapeutic options will clearly be fostered by the establishment of animal disease models that accurately simulate the patient's situation and the use of these disease models in 'next-generation' high-throughput small compound screens to define treatment options tailored to individual needs. To define suitable targets for therapeutic modulation, systems biology approaches that study complex biological systems as an integrated whole will pave the way to successful in vivo disease modeling and future drug discovery.
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