转录激活样效应因子的设计、构建及应用

Design, Construction, and Application of Transcription Activation-Like Effectors.

作者信息

Deng Peter, Carter Sakereh, Fink Kyle

机构信息

Stem Cell Program and Institute for Regenerative Cures, University of California, Davis, Sacramento, CA, USA.

Genome Center, MIND Institute, and Biochemistry and Molecular Medicine, University of California, Davis, Davis, CA, USA.

出版信息

Methods Mol Biol. 2019;1937:47-58. doi: 10.1007/978-1-4939-9065-8_3.

DOI:10.1007/978-1-4939-9065-8_3

PMID:30706389

Abstract

Transcription activator-like effectors (TALEs) are modular proteins derived from the plant Xanthomonas sp. pathogen that can be designed to target unique DNA sequences following a simple cipher. Customized TALE proteins can be used in a variety of molecular applications that include gene editing and transcriptional modulation. Presently, we provide a brief primer on the design and construction of TALEs. TALE proteins can be fused to a variety of different effector domains that alter the function of the TALE upon binding. This flexibility of TALE design and downstream effect may offer therapeutic applications that are discussed in this section. Finally, we provide a future perspective on TALE technology and what challenges remain for successful translation of gene-editing strategies to the clinic.

摘要

转录激活样效应因子（TALEs）是源自植物黄单胞菌属病原体的模块化蛋白质，按照一种简单的编码规则，可将其设计用于靶向独特的DNA序列。定制的TALE蛋白可用于包括基因编辑和转录调控在内的多种分子应用。目前，我们提供关于TALEs设计与构建的简要入门知识。TALE蛋白可与多种不同的效应结构域融合，这些结构域在结合后会改变TALE的功能。TALE设计的这种灵活性及其下游效应可能会带来本节所讨论的治疗应用。最后，我们对TALE技术的未来前景以及将基因编辑策略成功转化至临床仍面临的挑战进行了展望。