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.
Liver diseases are one of the leading causes of mortalityin the world. The hepatic illnesses, which includeinherited metabolic disorders, hemophilias and viralhepatitides, are complex and currently difficult to treat.The maturation of gene therapy has heralded newavenues for developing effective intervention for thesediseases. DNA modification using gene therapy is nowpossible and available technology may be exploitedto achieve long term therapeutic benefit. The abilityto edit DNA sequences specifically is of paramountimportance to advance gene therapy for application toliver diseases. Recent development of technologies thatallow for this has resulted in rapid advancement of genetherapy to treat several chronic illnesses. Improvementsin application of derivatives of zinc finger proteins （ZFPs）,transcription activator-like effectors （TALEs）, homingendonucleases （HEs） and clustered regularly interspacedpalindromic repeats （CRISPR） and CRISPR associated（Cas） systems have been particularly important. Thesesequence-specific technologies may be used to modifygenes permanently and also to alter gene transcriptionfor therapeutic purposes. This review describes progressin development of ZFPs, TALEs, HEs and CRISPR/Casfor application to treating liver diseases.
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
