Engineering synthetic TALE and CRISPR/Cas9 transcription factors for regulating gene expression.

Journal Article (Journal Article)

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.

Full Text

Duke Authors

Cited Authors

  • Kabadi, AM; Gersbach, CA

Published Date

  • September 2014

Published In

Volume / Issue

  • 69 / 2

Start / End Page

  • 188 - 197

PubMed ID

  • 25010559

Pubmed Central ID

  • PMC4175060

Electronic International Standard Serial Number (EISSN)

  • 1095-9130

International Standard Serial Number (ISSN)

  • 1046-2023

Digital Object Identifier (DOI)

  • 10.1016/j.ymeth.2014.06.014


  • eng