Literature DB >> 25271723

Expanding the genetic editing tool kit: ZFNs, TALENs, and CRISPR-Cas9.

Rajat M Gupta, Kiran Musunuru.   

Abstract

The past decade has been one of rapid innovation in genome-editing technology. The opportunity now exists for investigators to manipulate virtually any gene in a diverse range of cell types and organisms with targeted nucleases designed with sequence-specific DNA-binding domains. The rapid development of the field has allowed for highly efficient, precise, and now cost-effective means by which to generate human and animal models of disease using these technologies. This review will outline the recent development of genome-editing technology, culminating with the use of CRISPR-Cas9 to generate novel mammalian models of disease. While the road to using this same technology for treatment of human disease is long, the pace of innovation over the past five years and early successes in model systems build anticipation for this prospect.

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Year:  2014        PMID: 25271723      PMCID: PMC4191047          DOI: 10.1172/JCI72992

Source DB:  PubMed          Journal:  J Clin Invest        ISSN: 0021-9738            Impact factor:   14.808


  93 in total

1.  Heritable gene targeting in the mouse and rat using a CRISPR-Cas system.

Authors:  Dali Li; Zhongwei Qiu; Yanjiao Shao; Yuting Chen; Yuting Guan; Meizhen Liu; Yongmei Li; Na Gao; Liren Wang; Xiaoling Lu; Yongxiang Zhao; Mingyao Liu
Journal:  Nat Biotechnol       Date:  2013-08       Impact factor: 54.908

2.  Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease.

Authors:  Seung Woo Cho; Sojung Kim; Jong Min Kim; Jin-Soo Kim
Journal:  Nat Biotechnol       Date:  2013-01-29       Impact factor: 54.908

3.  A library of TAL effector nucleases spanning the human genome.

Authors:  Yongsub Kim; Jiyeon Kweon; Annie Kim; Jae Kyung Chon; Ji Yeon Yoo; Hye Joo Kim; Sojung Kim; Choongil Lee; Euihwan Jeong; Eugene Chung; Doyoung Kim; Mi Seon Lee; Eun Mi Go; Hye Jung Song; Hwangbeom Kim; Namjin Cho; Duhee Bang; Seokjoong Kim; Jin-Soo Kim
Journal:  Nat Biotechnol       Date:  2013-02-17       Impact factor: 54.908

4.  Direct production of mouse disease models by embryo microinjection of TALENs and oligodeoxynucleotides.

Authors:  Benedikt Wefers; Melanie Meyer; Oskar Ortiz; Martin Hrabé de Angelis; Jens Hansen; Wolfgang Wurst; Ralf Kühn
Journal:  Proc Natl Acad Sci U S A       Date:  2013-02-20       Impact factor: 11.205

5.  Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases.

Authors:  Elena E Perez; Jianbin Wang; Jeffrey C Miller; Yann Jouvenot; Kenneth A Kim; Olga Liu; Nathaniel Wang; Gary Lee; Victor V Bartsevich; Ya-Li Lee; Dmitry Y Guschin; Igor Rupniewski; Adam J Waite; Carmine Carpenito; Richard G Carroll; Jordan S Orange; Fyodor D Urnov; Edward J Rebar; Dale Ando; Philip D Gregory; James L Riley; Michael C Holmes; Carl H June
Journal:  Nat Biotechnol       Date:  2008-06-29       Impact factor: 54.908

6.  Genetic correction of a LRRK2 mutation in human iPSCs links parkinsonian neurodegeneration to ERK-dependent changes in gene expression.

Authors:  Peter Reinhardt; Benjamin Schmid; Lena F Burbulla; David C Schöndorf; Lydia Wagner; Michael Glatza; Susanne Höing; Gunnar Hargus; Susanna A Heck; Ashutosh Dhingra; Guangming Wu; Stephan Müller; Kathrin Brockmann; Torsten Kluba; Martina Maisel; Rejko Krüger; Daniela Berg; Yaroslav Tsytsyura; Cora S Thiel; Olympia-Ekaterini Psathaki; Jürgen Klingauf; Tanja Kuhlmann; Marlene Klewin; Heiko Müller; Thomas Gasser; Hans R Schöler; Jared Sterneckert
Journal:  Cell Stem Cell       Date:  2013-03-07       Impact factor: 24.633

7.  Insertion of DNA sequences into the human chromosomal beta-globin locus by homologous recombination.

Authors:  O Smithies; R G Gregg; S S Boggs; M A Koralewski; R S Kucherlapati
Journal:  Nature       Date:  1985 Sep 19-25       Impact factor: 49.962

8.  One-step generation of mice carrying mutations in multiple genes by CRISPR/Cas-mediated genome engineering.

Authors:  Haoyi Wang; Hui Yang; Chikdu S Shivalila; Meelad M Dawlaty; Albert W Cheng; Feng Zhang; Rudolf Jaenisch
Journal:  Cell       Date:  2013-05-02       Impact factor: 41.582

9.  Differential integrity of TALE nuclease genes following adenoviral and lentiviral vector gene transfer into human cells.

Authors:  Maarten Holkers; Ignazio Maggio; Jin Liu; Josephine M Janssen; Francesca Miselli; Claudio Mussolino; Alessandra Recchia; Toni Cathomen; Manuel A F V Gonçalves
Journal:  Nucleic Acids Res       Date:  2012-12-28       Impact factor: 16.971

10.  Highly active zinc-finger nucleases by extended modular assembly.

Authors:  Mital S Bhakta; Isabelle M Henry; David G Ousterout; Kumitaa Theva Das; Sarah H Lockwood; Joshua F Meckler; Mark C Wallen; Artem Zykovich; Yawei Yu; Heather Leo; Lifeng Xu; Charles A Gersbach; David J Segal
Journal:  Genome Res       Date:  2012-12-05       Impact factor: 9.043

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  121 in total

Review 1.  Human Germline Genome Editing.

Authors:  Kelly E Ormond; Douglas P Mortlock; Derek T Scholes; Yvonne Bombard; Lawrence C Brody; W Andrew Faucett; Nanibaa' A Garrison; Laura Hercher; Rosario Isasi; Anna Middleton; Kiran Musunuru; Daniel Shriner; Alice Virani; Caroline E Young
Journal:  Am J Hum Genet       Date:  2017-08-03       Impact factor: 11.025

Review 2.  Neurology--the next 10 years.

Authors:  Ralf Baron; Donna M Ferriero; Giovanni B Frisoni; Chetan Bettegowda; Ziya L Gokaslan; John A Kessler; Annamaria Vezzani; Stephen G Waxman; Sven Jarius; Brigitte Wildemann; Michael Weller
Journal:  Nat Rev Neurol       Date:  2015-10-27       Impact factor: 42.937

3.  Self-cleaving ribozymes enable the production of guide RNAs from unlimited choices of promoters for CRISPR/Cas9 mediated genome editing.

Authors:  Yubing He; Tao Zhang; Ning Yang; Meilian Xu; Lang Yan; Lihao Wang; Rongchen Wang; Yunde Zhao
Journal:  J Genet Genomics       Date:  2017-08-24       Impact factor: 4.275

4.  Synthetic microbial consortia for biosynthesis and biodegradation: promises and challenges.

Authors:  Shun Che; Yujie Men
Journal:  J Ind Microbiol Biotechnol       Date:  2019-07-05       Impact factor: 3.346

Review 5.  Cure for thalassemia major - from allogeneic hematopoietic stem cell transplantation to gene therapy.

Authors:  Alok Srivastava; Ramachandran V Shaji
Journal:  Haematologica       Date:  2016-12-01       Impact factor: 9.941

6.  Efficient, footprint-free human iPSC genome editing by consolidation of Cas9/CRISPR and piggyBac technologies.

Authors:  Gang Wang; Luhan Yang; Dennis Grishin; Xavier Rios; Lillian Y Ye; Yong Hu; Kai Li; Donghui Zhang; George M Church; William T Pu
Journal:  Nat Protoc       Date:  2016-12-08       Impact factor: 13.491

Review 7.  Genome editing in cardiovascular diseases.

Authors:  Alanna Strong; Kiran Musunuru
Journal:  Nat Rev Cardiol       Date:  2016-09-09       Impact factor: 32.419

Review 8.  In vivo epigenome editing and transcriptional modulation using CRISPR technology.

Authors:  Cia-Hin Lau; Yousin Suh
Journal:  Transgenic Res       Date:  2018-10-04       Impact factor: 2.788

Review 9.  Society for Pediatric Research 2015 Young Investigator Award: genetics of human hematopoiesis-what patients can teach us about blood cell production.

Authors:  Aoi Wakabayashi; Vijay G Sankaran
Journal:  Pediatr Res       Date:  2015-11-17       Impact factor: 3.756

Review 10.  Zebrafish as a disease model for studying human hepatocellular carcinoma.

Authors:  Jeng-Wei Lu; Yi-Jung Ho; Yi-Ju Yang; Heng-An Liao; Shih-Ci Ciou; Liang-In Lin; Da-Liang Ou
Journal:  World J Gastroenterol       Date:  2015-11-14       Impact factor: 5.742

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