Literature DB >> 28895847

Therapeutic Gene Editing Safety and Specificity.

Christopher T Lux1, Andrew M Scharenberg2.   

Abstract

Therapeutic gene editing is significant for medical advancement. Safety is intricately linked to the specificity of the editing tools used to cut at precise genomic targets. Improvements can be achieved by thoughtful design of nucleases and repair templates, analysis of off-target editing, and careful utilization of viral vectors. Advancements in DNA repair mechanisms and development of new generations of tools improve targeting of specific sequences while minimizing risks. It is important to plot a safe course for future clinical trials. This article reviews safety and specificity for therapeutic gene editing to spur dialogue and advancement.
Copyright © 2017 Elsevier Inc. All rights reserved.

Entities:  

Keywords:  Gene editing; Gene therapy; Off-target; Safety; Specific

Mesh：

Year:  2017        PMID： 28895847      PMCID： PMC5653273          DOI： 10.1016/j.hoc.2017.05.002

Source DB:  PubMed          Journal:  Hematol Oncol Clin North Am        ISSN： 0889-8588            Impact factor:   3.722


  29 in total

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Authors:  John van der Oost; Matthijs M Jore; Edze R Westra; Magnus Lundgren; Stan J J Brouns
Journal:  Trends Biochem Sci       Date:  2009-07-29       Impact factor: 13.807

2.  Improved specificity of TALE-based genome editing using an expanded RVD repertoire.

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Journal:  Nat Methods       Date:  2015-03-23       Impact factor: 28.547

Review 3.  TALEN-mediated genome editing: prospects and perspectives.

Authors:  David A Wright; Ting Li; Bing Yang; Martin H Spalding
Journal:  Biochem J       Date:  2014-08-15       Impact factor: 3.857

4.  CRISPR/Cas9-mediated genome engineering: an adeno-associated viral (AAV) vector toolbox.

Authors:  Elena Senís; Chronis Fatouros; Stefanie Große; Ellen Wiedtke; Dominik Niopek; Ann-Kristin Mueller; Kathleen Börner; Dirk Grimm
Journal:  Biotechnol J       Date:  2014-10-06       Impact factor: 4.677

5.  Genome-wide translocation sequencing reveals mechanisms of chromosome breaks and rearrangements in B cells.

Authors:  Roberto Chiarle; Yu Zhang; Richard L Frock; Susanna M Lewis; Benoit Molinie; Yu-Jui Ho; Darienne R Myers; Vivian W Choi; Mara Compagno; Daniel J Malkin; Donna Neuberg; Stefano Monti; Cosmas C Giallourakis; Monica Gostissa; Frederick W Alt
Journal:  Cell       Date:  2011-09-30       Impact factor: 41.582

6.  Efficient design and assembly of custom TALEN and other TAL effector-based constructs for DNA targeting.

Authors:  Tomas Cermak; Erin L Doyle; Michelle Christian; Li Wang; Yong Zhang; Clarice Schmidt; Joshua A Baller; Nikunj V Somia; Adam J Bogdanove; Daniel F Voytas
Journal:  Nucleic Acids Res       Date:  2011-04-14       Impact factor: 16.971

7.  GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases.

Authors:  Shengdar Q Tsai; Zongli Zheng; Nhu T Nguyen; Matthew Liebers; Ved V Topkar; Vishal Thapar; Nicolas Wyvekens; Cyd Khayter; A John Iafrate; Long P Le; Martin J Aryee; J Keith Joung
Journal:  Nat Biotechnol       Date:  2014-12-16       Impact factor: 54.908

Review 8.  Improving the safety of cell therapy products by suicide gene transfer.

Authors:  Benjamin S Jones; Lawrence S Lamb; Frederick Goldman; Antonio Di Stasi
Journal:  Front Pharmacol       Date:  2014-11-27       Impact factor: 5.810

9.  megaTALs: a rare-cleaving nuclease architecture for therapeutic genome engineering.

Authors:  Sandrine Boissel; Jordan Jarjour; Alexander Astrakhan; Andrew Adey; Agnès Gouble; Philippe Duchateau; Jay Shendure; Barry L Stoddard; Michael T Certo; David Baker; Andrew M Scharenberg
Journal:  Nucleic Acids Res       Date:  2013-11-26       Impact factor: 16.971

10.  CRISPR/Cas9 systems targeting β-globin and CCR5 genes have substantial off-target activity.

Authors:  Thomas J Cradick; Eli J Fine; Christopher J Antico; Gang Bao
Journal:  Nucleic Acids Res       Date:  2013-08-11       Impact factor: 16.971
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  6 in total

1.  Influence of gene modification in biological behaviors and responses of mouse lung telocytes to inflammation.

Authors:  Dongli Song; Menglin Xu; Ruixue Qi; Ruihua Ma; Yile Zhou; Duojiao Wu; Hao Fang; Xiangdong Wang
Journal:  J Transl Med       Date:  2019-05-15       Impact factor: 5.531

2.  Systemic mRNA Therapy for the Treatment of Fabry Disease: Preclinical Studies in Wild-Type Mice, Fabry Mouse Model, and Wild-Type Non-human Primates.

Authors:  Xuling Zhu; Ling Yin; Matt Theisen; Jenny Zhuo; Summar Siddiqui; Becca Levy; Vladimir Presnyak; Andrea Frassetto; Jaclyn Milton; Timothy Salerno; Kerry E Benenato; Joe Milano; Andy Lynn; Staci Sabnis; Kristine Burke; Gilles Besin; Christine M Lukacs; Lin T Guey; Patrick F Finn; Paolo G V Martini
Journal:  Am J Hum Genet       Date:  2019-03-14       Impact factor: 11.025

Review 3.  Sphingosine phosphate lyase insufficiency syndrome (SPLIS): A novel inborn error of sphingolipid metabolism.

Authors:  Youn-Jeong Choi; Julie D Saba
Journal:  Adv Biol Regul       Date:  2018-09-25

4.  Comprehensive assessment of miniature CRISPR-Cas12f nucleases for gene disruption.

Authors:  Changchang Xin; Jianhang Yin; Shaopeng Yuan; Liqiong Ou; Mengzhu Liu; Weiwei Zhang; Jiazhi Hu
Journal:  Nat Commun       Date:  2022-09-24       Impact factor: 17.694

Review 5.  Managing Bardet-Biedl Syndrome-Now and in the Future.

Authors:  Elizabeth Forsythe; Joanna Kenny; Chiara Bacchelli; Philip L Beales
Journal:  Front Pediatr       Date:  2018-02-13       Impact factor: 3.418

6.  Reprogramming human T cell function and specificity with non-viral genome targeting.

Authors:  Theodore L Roth; Cristina Puig-Saus; Ruby Yu; Eric Shifrut; Julia Carnevale; P Jonathan Li; Joseph Hiatt; Justin Saco; Paige Krystofinski; Han Li; Victoria Tobin; David N Nguyen; Michael R Lee; Amy L Putnam; Andrea L Ferris; Jeff W Chen; Jean-Nicolas Schickel; Laurence Pellerin; David Carmody; Gorka Alkorta-Aranburu; Daniela Del Gaudio; Hiroyuki Matsumoto; Montse Morell; Ying Mao; Min Cho; Rolen M Quadros; Channabasavaiah B Gurumurthy; Baz Smith; Michael Haugwitz; Stephen H Hughes; Jonathan S Weissman; Kathrin Schumann; Jonathan H Esensten; Andrew P May; Alan Ashworth; Gary M Kupfer; Siri Atma W Greeley; Rosa Bacchetta; Eric Meffre; Maria Grazia Roncarolo; Neil Romberg; Kevan C Herold; Antoni Ribas; Manuel D Leonetti; Alexander Marson
Journal:  Nature       Date:  2018-07-11       Impact factor: 49.962
  6 in total

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