Open AccessRecent advances in the use of ZFN-mediated gene editing for human gene therapy
Author(s) -
Srinivasan Chandrasegaran
Publication year - 2017
Publication title -
cell and gene therapy insights
Language(s) - English
Resource type - Journals
eISSN - 2397-0545
pISSN - 2059-7800
DOI - 10.18609/cgti.2017.005
Subject(s) - zinc finger nuclease , genome editing , transcription activator like effector nuclease , genetic enhancement , human genome , gene , computational biology , genome engineering , biology , zinc finger , genome , genetics , transcription factor
Targeted genome editing with programmable nucleases has revolutionized biomedical research. The ability to make site-specific modifications to the human genome, has invoked a paradigm shift in gene therapy. Using gene editing technologies, the sequence in the human genome can now be precisely engineered to achieve a therapeutic effect. Zinc finger nucleases (ZFNs) were the first programmable nucleases designed to target and cleave custom sites. This article summarizes the advances in the use of ZFN-mediated gene editing for human gene therapy and discusses the challenges associated with translating this gene editing technology into clinical use.