Genome-editing technologies: Principles and applications

Thomas Gaj, Shannon J. Sirk, Sai Lan Shui, Jia Liu

Research output: Contribution to journalArticle

Abstract

Targeted nucleases have provided researchers with the ability to manipulate virtually any genomic sequence, enabling the facile creation of isogenic cell lines and animal models for the study of human disease, and promoting exciting new possibilities for human gene therapy. Here we review three foundational technologies—clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated protein 9 (Cas9), transcription activator- like effector nucleases (TALENs), and zinc-finger nucleases (ZFNs). We discuss the engineering advances that facilitated their development and highlight several achievements in genome engineering that were made possible by these tools. We also consider artificial transcription factors, illustrating howthis technology can complement targeted nucleases for synthetic biology and gene therapy.

Original languageEnglish (US)
Article numbera023754
JournalCold Spring Harbor Perspectives in Biology
Volume8
Issue number12
DOIs
StatePublished - Jan 1 2016
Externally publishedYes

ASJC Scopus subject areas

  • Biochemistry, Genetics and Molecular Biology(all)

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