Genome-editing technologies: Principles and applications

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

Research output: Contribution to journalArticlepeer-review


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
Issue number12
StatePublished - 2016
Externally publishedYes

ASJC Scopus subject areas

  • General Biochemistry, Genetics and Molecular Biology


Dive into the research topics of 'Genome-editing technologies: Principles and applications'. Together they form a unique fingerprint.

Cite this