期刊
METHODS
卷 69, 期 2, 页码 188-197出版社
ACADEMIC PRESS INC ELSEVIER SCIENCE
DOI: 10.1016/j.ymeth.2014.06.014
关键词
Gene regulation; Transcription factor; Protein engineering; TAL effector; TALE; CRISPR; Cas9; Gene therapy; Genetic reprogramming; Synthetic biology
资金
- US National Institutes of Health (NIH) Director's New Innovator Award [DP2OD008586]
- National Science Foundation (NSF) Faculty Early Career Development (CAREER) Award [CBET-1151035]
- NIH [R01DA036865, R21AR065956, R03AR061042]
- Muscular Dystrophy Association [MDA277360]
- Nancy Taylor Foundation
- The Hartwell Foundation
- March of Dimes
- American Heart Association Scientist Development Grant [10SDG3060033]
- Div Of Chem, Bioeng, Env, & Transp Sys
- Directorate For Engineering [1151035] Funding Source: National Science Foundation
Engineered DNA-binding proteins that can be targeted to specific sites in the genome to manipulate gene expression have enabled many advances in biomedical research. This includes generating tools to study fundamental aspects of gene regulation and the development of a new class of gene therapies that alter the expression of endogenous genes. Designed transcription factors have entered clinical trials for the treatment of human diseases and others are in preclinical development. High-throughput and user-friendly platforms for designing synthetic DNA-binding proteins present innovative methods for deciphering cell biology and designing custom synthetic gene circuits. We review two platforms for designing synthetic transcription factors for manipulating gene expression: Transcription activator-like effectors (TALEs) and the RNA-guided clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 system. We present an overview of each technology and a guide for designing and assembling custom TALE-and CRISPR/Cas9-based transcription factors. We also discuss characteristics of each platform that are best suited for different applications. (C) 2014 Elsevier Inc. All rights reserved.
作者
我是这篇论文的作者
点击您的名字以认领此论文并将其添加到您的个人资料中。
推荐
暂无数据