Literature DB >> 28487442

CRISPR/Cas9: at the cutting edge of hepatology.

Francis P Pankowicz1,2, Kelsey E Jarrett3,4, William R Lagor1,3,4,5, Karl-Dimiter Bissig1,2,5,6,7,8,9.   

Abstract

Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas9 genome engineering has revolutionised biomedical science and we are standing on the cusp of medical transformation. The therapeutic potential of this technology is tremendous, however, its translation to the clinic will be challenging. In this article, we review recent progress using this genome editing technology and explore its potential uses in studying and treating diseases of the liver. We discuss the development of new research tools and animal models as well as potential clinical applications, strategies and challenges. Published by the BMJ Publishing Group Limited. For permission to use (where not already granted under a licence) please go to http://www.bmj.com/company/products-services/rights-and-licensing/.

Entities:  

Keywords:  GENE TARGETING; GENE THERAPY; GENE TRANSFER; HEPATOCYTE

Mesh:

Year:  2017        PMID: 28487442      PMCID: PMC5878048          DOI: 10.1136/gutjnl-2016-313565

Source DB:  PubMed          Journal:  Gut        ISSN: 0017-5749            Impact factor:   23.059


  179 in total

1.  Identification of genes that are associated with DNA repeats in prokaryotes.

Authors:  Ruud Jansen; Jan D A van Embden; Wim Gaastra; Leo M Schouls
Journal:  Mol Microbiol       Date:  2002-03       Impact factor: 3.501

Review 2.  Regulation of DNA repair throughout the cell cycle.

Authors:  Dana Branzei; Marco Foiani
Journal:  Nat Rev Mol Cell Biol       Date:  2008-02-20       Impact factor: 94.444

3.  Long-range disruption of gene expression by a selectable marker cassette.

Authors:  C T Pham; D M MacIvor; B A Hug; J W Heusel; T J Ley
Journal:  Proc Natl Acad Sci U S A       Date:  1996-11-12       Impact factor: 11.205

4.  Generation of gene-modified mice via Cas9/RNA-mediated gene targeting.

Authors:  Bin Shen; Jun Zhang; Hongya Wu; Jianying Wang; Ke Ma; Zheng Li; Xueguang Zhang; Pumin Zhang; Xingxu Huang
Journal:  Cell Res       Date:  2013-04-02       Impact factor: 25.617

Review 5.  Molecular biology of PCSK9: its role in LDL metabolism.

Authors:  Jay D Horton; Jonathan C Cohen; Helen H Hobbs
Journal:  Trends Biochem Sci       Date:  2007-01-09       Impact factor: 13.807

Review 6.  Defining and improving the genome-wide specificities of CRISPR-Cas9 nucleases.

Authors:  Shengdar Q Tsai; J Keith Joung
Journal:  Nat Rev Genet       Date:  2016-05       Impact factor: 53.242

7.  Adeno-associated virus vectors integrate at chromosome breakage sites.

Authors:  Daniel G Miller; Lisa M Petek; David W Russell
Journal:  Nat Genet       Date:  2004-06-20       Impact factor: 38.330

8.  Phage response to CRISPR-encoded resistance in Streptococcus thermophilus.

Authors:  Hélène Deveau; Rodolphe Barrangou; Josiane E Garneau; Jessica Labonté; Christophe Fremaux; Patrick Boyaval; Dennis A Romero; Philippe Horvath; Sylvain Moineau
Journal:  J Bacteriol       Date:  2007-12-07       Impact factor: 3.490

9.  Development and rescue of human familial hypercholesterolaemia in a xenograft mouse model.

Authors:  Beatrice Bissig-Choisat; Lili Wang; Xavier Legras; Pradip K Saha; Leon Chen; Peter Bell; Francis P Pankowicz; Matthew C Hill; Mercedes Barzi; Claudia Kettlun Leyton; Hon-Chiu Eastwood Leung; Robert L Kruse; Ryan W Himes; John A Goss; James M Wilson; Lawrence Chan; William R Lagor; Karl-Dimiter Bissig
Journal:  Nat Commun       Date:  2015-06-17       Impact factor: 14.919

10.  Genome editing with RNA-guided Cas9 nuclease in zebrafish embryos.

Authors:  Nannan Chang; Changhong Sun; Lu Gao; Dan Zhu; Xiufei Xu; Xiaojun Zhu; Jing-Wei Xiong; Jianzhong Jeff Xi
Journal:  Cell Res       Date:  2013-03-26       Impact factor: 25.617

View more
  16 in total

1.  CRISPR-Cas-related technologies in basic and translational liver research.

Authors:  Chun-Qing Song; Wen Xue
Journal:  Nat Rev Gastroenterol Hepatol       Date:  2018-02-14       Impact factor: 46.802

2.  In Vivo Gene Editing in Lipid and Atherosclerosis Research.

Authors:  Marco De Giorgi; Kelsey E Jarrett; Thomas Q de Aguiar Vallim; William R Lagor
Journal:  Methods Mol Biol       Date:  2022

Review 3.  Biofabrication of Autologous Human Hepatocytes for Transplantation: How Do We Get There?

Authors:  Nandini Agarwal; Branimir Popovic; Nicole J Martucci; Nicolas A Fraunhoffer; Alejandro Soto-Gutierrez
Journal:  Gene Expr       Date:  2018-08-24

4.  Use of the CRISPR/Cas9-based epigenetic gene activation system In Vivo: A new potential therapeutic modality.

Authors:  Samar H Ibrahim; Keith D Robertson
Journal:  Hepatology       Date:  2018-05-14       Impact factor: 17.425

Review 5.  The Promise and Challenge of In Vivo Delivery for Genome Therapeutics.

Authors:  Ross C Wilson; Luke A Gilbert
Journal:  ACS Chem Biol       Date:  2017-10-19       Impact factor: 5.100

6.  Correction of metabolic abnormalities in a mouse model of glycogen storage disease type Ia by CRISPR/Cas9-based gene editing.

Authors:  Irina Arnaoutova; Lisa Zhang; Hung-Dar Chen; Brian C Mansfield; Janice Y Chou
Journal:  Mol Ther       Date:  2020-12-23       Impact factor: 11.454

7.  MicroRNA 130a Regulates both Hepatitis C Virus and Hepatitis B Virus Replication through a Central Metabolic Pathway.

Authors:  Wenyu Lin; Raymond T Chung; Xiaoqiong Duan; Shilin Li; Jacinta A Holmes; Zeng Tu; Yujia Li; Dachuan Cai; Xiao Liu; Wenting Li; Chunhui Yang; Baihai Jiao; Esperance A Schaefer; Dahlene N Fusco; Shadi Salloum; Limin Chen
Journal:  J Virol       Date:  2018-03-14       Impact factor: 5.103

8.  Targeting the Apoa1 locus for liver-directed gene therapy.

Authors:  Marco De Giorgi; Ang Li; Ayrea Hurley; Mercedes Barzi; Alexandria M Doerfler; Nikitha A Cherayil; Harrison E Smith; Jonathan D Brown; Charles Y Lin; Karl-Dimiter Bissig; Gang Bao; William R Lagor
Journal:  Mol Ther Methods Clin Dev       Date:  2021-04-24       Impact factor: 6.698

Review 9.  Novel vectors and approaches for gene therapy in liver diseases.

Authors:  Sheila Maestro; Nicholas D Weber; Nerea Zabaleta; Rafael Aldabe; Gloria Gonzalez-Aseguinolaza
Journal:  JHEP Rep       Date:  2021-04-30

Review 10.  CRISPR-Cas Targeting of Host Genes as an Antiviral Strategy.

Authors:  Shuliang Chen; Xiao Yu; Deyin Guo
Journal:  Viruses       Date:  2018-01-16       Impact factor: 5.048

View more

北京卡尤迪生物科技股份有限公司 © 2022-2023.