Progress and prospects of engineered sequence-specific DNA modulating technologies for the management of liver diseases

作     者：Samantha A Nicholson Buhle Moyo Patrick B Arbuthnot 

作者机构：Wits/SA MRC Antiviral Gene Therapy Research Unit School of Pathology Health Sciences Faculty University of the Witwatersrand 

基　　金：The South African National Research Foundation(NRF,GUNs 81768,81692,68339,85981 and 77954) Poliomyelitis Research Foundation Claude Leon Foundation(SAN) The University of the Witwatersrand Research Council(BM)and Medical Research Council 

出 版 物：《World Journal of Hepatology》 (世界肝病学杂志（英文版）（电子版）)

年 卷 期：2015年第7卷第6期

页      码：859-873页

摘      要：Liver diseases are one of the leading causes of mortality in the world. The hepatic illnesses, which include inherited metabolic disorders, hemophilias and viralhepatitides, 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.

主 题 词：Gene therapy Zinc fingers Transcriptionactivator-like effectors Clustered regularly interspacedshort palindromic repeats Homing endonucleases Liver diseases 

学科分类：1002[医学-临床医学类] 100201[100201] 10[医学] 

D　O　I：10.4254/wjh.v7.i6.859

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