Literature DB >> 29266637

CRISPR/Cas9 therapeutics for liver diseases.

Rajagopal N Aravalli1, Clifford J Steer2,3.   

Abstract

The development of innovative genome editing techniques in recent years has revolutionized the field of biomedicine. Among the novel approaches, the clustered regularly interspaced short palindromic repeat/CRISPR-associated protein (CRISPR/Cas9) technology has become the most popular, in part due to its matchless ability to carry out gene editing at the target site with great precision. With considerable successes in animal and preclinical studies, CRISPR/Cas9-mediated gene editing has paved the way for its use in human trials, including patients with a variety of liver diseases. Gene editing is a logical therapeutic approach for liver diseases because many metabolic and acquired disorders are caused by mutations within a single gene. In this review, we provide an overview on current and emerging therapeutic strategies for the treatment of liver diseases using the CRISPR/Cas9 technology.
© 2017 Wiley Periodicals, Inc.

Entities:  

Keywords:  CRISPR; gene editing; gene therapy; liver, non-viral vectors; viral vectors

Mesh:

Year:  2018        PMID: 29266637     DOI: 10.1002/jcb.26627

Source DB:  PubMed          Journal:  J Cell Biochem        ISSN: 0730-2312            Impact factor:   4.429


  4 in total

Review 1.  State-of-the-Art 2019 on Gene Therapy for Phenylketonuria.

Authors:  Hiu Man Grisch-Chan; Gerald Schwank; Cary O Harding; Beat Thöny
Journal:  Hum Gene Ther       Date:  2019-09-09       Impact factor: 5.695

2.  LRP6-CRISPR prevents activation of hepatic stellate cells and liver fibrogenesis in rats.

Authors:  Linghua Yu; Linlin Wang; Huixing Yi; Xiaojun Wu
Journal:  Am J Transl Res       Date:  2020-02-15       Impact factor: 4.060

3.  Low-density lipoprotein receptor-deficient hepatocytes differentiated from induced pluripotent stem cells allow familial hypercholesterolemia modeling, CRISPR/Cas-mediated genetic correction, and productive hepatitis C virus infection.

Authors:  Jérôme Caron; Véronique Pène; Laia Tolosa; Maxime Villaret; Eléanor Luce; Angélique Fourrier; Jean-Marie Heslan; Samir Saheb; Eric Bruckert; María José Gómez-Lechón; Tuan Huy Nguyen; Arielle R Rosenberg; Anne Weber; Anne Dubart-Kupperschmitt
Journal:  Stem Cell Res Ther       Date:  2019-07-29       Impact factor: 6.832

4.  Exosome-mediated delivery of Cas9 ribonucleoprotein complexes for tissue-specific gene therapy of liver diseases.

Authors:  Tao Wan; Jiafeng Zhong; Qi Pan; Tianhua Zhou; Yuan Ping; Xiangrui Liu
Journal:  Sci Adv       Date:  2022-09-14       Impact factor: 14.957

  4 in total

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