Published online Apr 28, 2015. doi: 10.4254/wjh.v7.i6.859
Peer-review started: September 13, 2014
First decision: September 28, 2014
Revised: December 16, 2014
Accepted: January 18, 2015
Article in press: January 20, 2015
Published online: April 28, 2015
Processing time: 232 Days and 3.2 Hours
Liver diseases are one of the leading causes of mortality in the world. The hepatic illnesses, which include inherited metabolic disorders, hemophilias and viral hepatitides, are complex and currently difficult to treat. The maturation of gene therapy has heralded new avenues for developing effective intervention for these diseases. DNA modification using gene therapy is now possible and available technology may be exploited to achieve long term therapeutic benefit. The ability to edit DNA sequences specifically is of paramount importance to advance gene therapy for application to liver diseases. Recent development of technologies that allow for this has resulted in rapid advancement of gene therapy to treat several chronic illnesses. Improvements in application of derivatives of zinc finger proteins (ZFPs), transcription activator-like effectors (TALEs), homing endonucleases (HEs) and clustered regularly interspaced palindromic repeats (CRISPR) and CRISPR associated (Cas) systems have been particularly important. These sequence-specific technologies may be used to modify genes permanently and also to alter gene transcription for therapeutic purposes. This review describes progress in development of ZFPs, TALEs, HEs and CRISPR/Cas for application to treating liver diseases.
Core tip: The treatment of liver diseases is varied and often complicated. Gene editing is being developed to treat a variety of chronic disorders and has exciting potential for curing hepatic diseases. Engineering of derivatives of zinc finger proteins, transcription activator-like effectors, homing endonucleases and clustered regularly interspaced palindromic repeats potentially enables sequence-specific gene editing. These DNA binding proteins may be used to alter genes permanently or to influence the epigenetic status of liver cells for therapeutic benefit.