Developmental history and application of CRISPR in human disease

Abstract
Genome editing tools are programmable artificial nucleases, mainly including zinc‐finger nucleases (ZFNs), transcription activator‐like effector nucleases (TALENs) and clustered regularly interspaced short palindromic repeat (CRISPR). By recognizing and cleaving specific DNA sequences, genome editing tools make it possible to generate site‐specific DNA double‐strand breaks (DSBs) in the genome. DSBs will then be repaired by either error‐prone non‐homologous end joining (NHEJ) or high‐fidelity homologous recombination (HR) mechanisms. Through these two different mechanisms, endogenous genes can be knocked out or precisely repaired/modified. Rapid developments in genome editing tools, especially CRISPR, have revolutionized human disease models generation; for example, various zebrafish, mouse, rat, pig, monkey and human cell lines have been constructed. Here, we review the developmental history of CRISPR and its application in studies of human diseases. In addition, we also briefly discussed the therapeutic application of CRISPR in the near future.

Source: The Journal of Gene Medicine - May 1, 2017 Category: Genetics & Stem Cells Authors: Puping Liang, Xiya Zhang, Yuxi Chen, Junjiu Huang Tags: REVIEW ARTICLE Source Type: research