Literature DB >> 24362028

A cut above the rest: targeted genome editing technologies in human pluripotent stem cells.

Mo Li1, Keiichiro Suzuki, Na Young Kim, Guang-Hui Liu, Juan Carlos Izpisua Belmonte.   

Abstract

Human pluripotent stem cells (hPSCs) offer unprecedented opportunities to study cellular differentiation and model human diseases. The ability to precisely modify any genomic sequence holds the key to realizing the full potential of hPSCs. Thanks to the rapid development of novel genome editing technologies driven by the enormous interest in the hPSC field, genome editing in hPSCs has evolved from being a daunting task a few years ago to a routine procedure in most laboratories. Here, we provide an overview of the mainstream genome editing tools, including zinc finger nucleases, transcription activator-like effector nucleases, clustered regularly interspaced short palindromic repeat/CAS9 RNA-guided nucleases, and helper-dependent adenoviral vectors. We discuss the features and limitations of these technologies, as well as how these factors influence the utility of these tools in basic research and therapies.

Entities:  

Keywords:  CRISPR/CAS9; Embryonic Stem Cell; Gene Therapy; Genome Editing; HDAdV; Induced Pluripotent Stem Cell (iPSC); Stem Cells; TALEN; ZFN

Mesh:

Substances:

Year:  2013        PMID: 24362028      PMCID: PMC3931021          DOI: 10.1074/jbc.R113.488247

Source DB:  PubMed          Journal:  J Biol Chem        ISSN: 0021-9258            Impact factor:   5.157


  61 in total

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Authors:  Mo Li; Keiichiro Suzuki; Jing Qu; Preeti Saini; Ilir Dubova; Fei Yi; Jungmin Lee; Ignacio Sancho-Martinez; Guang-Hui Liu; Juan Carlos Izpisua Belmonte
Journal:  Cell Res       Date:  2011-11-22       Impact factor: 25.617

Review 2.  RNA-guided genetic silencing systems in bacteria and archaea.

Authors:  Blake Wiedenheft; Samuel H Sternberg; Jennifer A Doudna
Journal:  Nature       Date:  2012-02-15       Impact factor: 49.962

3.  Generation of isogenic pluripotent stem cells differing exclusively at two early onset Parkinson point mutations.

Authors:  Frank Soldner; Josée Laganière; Albert W Cheng; Dirk Hockemeyer; Qing Gao; Raaji Alagappan; Vikram Khurana; Lawrence I Golbe; Richard H Myers; Susan Lindquist; Lei Zhang; Dmitry Guschin; Lauren K Fong; B Joseph Vu; Xiangdong Meng; Fyodor D Urnov; Edward J Rebar; Philip D Gregory; H Steve Zhang; Rudolf Jaenisch
Journal:  Cell       Date:  2011-07-14       Impact factor: 41.582

4.  An unbiased genome-wide analysis of zinc-finger nuclease specificity.

Authors:  Richard Gabriel; Angelo Lombardo; Anne Arens; Jeffrey C Miller; Pietro Genovese; Christine Kaeppel; Ali Nowrouzi; Cynthia C Bartholomae; Jianbin Wang; Geoffrey Friedman; Michael C Holmes; Philip D Gregory; Hanno Glimm; Manfred Schmidt; Luigi Naldini; Christof von Kalle
Journal:  Nat Biotechnol       Date:  2011-08-07       Impact factor: 54.908

5.  Editing T cell specificity towards leukemia by zinc finger nucleases and lentiviral gene transfer.

Authors:  Elena Provasi; Pietro Genovese; Angelo Lombardo; Zulma Magnani; Pei-Qi Liu; Andreas Reik; Victoria Chu; David E Paschon; Lei Zhang; Jurgen Kuball; Barbara Camisa; Attilio Bondanza; Giulia Casorati; Maurilio Ponzoni; Fabio Ciceri; Claudio Bordignon; Philip D Greenberg; Michael C Holmes; Philip D Gregory; Luigi Naldini; Chiara Bonini
Journal:  Nat Med       Date:  2012-05       Impact factor: 53.440

6.  The crystal structure of TAL effector PthXo1 bound to its DNA target.

Authors:  Amanda Nga-Sze Mak; Philip Bradley; Raul A Cernadas; Adam J Bogdanove; Barry L Stoddard
Journal:  Science       Date:  2012-01-05       Impact factor: 47.728

7.  High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells.

Authors:  Yanfang Fu; Jennifer A Foden; Cyd Khayter; Morgan L Maeder; Deepak Reyon; J Keith Joung; Jeffry D Sander
Journal:  Nat Biotechnol       Date:  2013-06-23       Impact factor: 54.908

8.  Revealing off-target cleavage specificities of zinc-finger nucleases by in vitro selection.

Authors:  Vikram Pattanayak; Cherie L Ramirez; J Keith Joung; David R Liu
Journal:  Nat Methods       Date:  2011-08-07       Impact factor: 28.547

9.  Targeted gene correction of α1-antitrypsin deficiency in induced pluripotent stem cells.

Authors:  Kosuke Yusa; S Tamir Rashid; Helene Strick-Marchand; Ignacio Varela; Pei-Qi Liu; David E Paschon; Elena Miranda; Adriana Ordóñez; Nicholas R F Hannan; Foad J Rouhani; Sylvie Darche; Graeme Alexander; Stefan J Marciniak; Noemi Fusaki; Mamoru Hasegawa; Michael C Holmes; James P Di Santo; David A Lomas; Allan Bradley; Ludovic Vallier
Journal:  Nature       Date:  2011-10-12       Impact factor: 49.962

10.  A novel TALE nuclease scaffold enables high genome editing activity in combination with low toxicity.

Authors:  Claudio Mussolino; Robert Morbitzer; Fabienne Lütge; Nadine Dannemann; Thomas Lahaye; Toni Cathomen
Journal:  Nucleic Acids Res       Date:  2011-08-03       Impact factor: 16.971

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  69 in total

Review 1.  Direct somatic lineage conversion.

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Journal:  Philos Trans R Soc Lond B Biol Sci       Date:  2015-10-19       Impact factor: 6.237

Review 2.  Understanding platelet generation from megakaryocytes: implications for in vitro-derived platelets.

Authors:  Xiuli Sim; Mortimer Poncz; Paul Gadue; Deborah L French
Journal:  Blood       Date:  2016-01-19       Impact factor: 22.113

Review 3.  Transcriptional regulation of GAD1 GABA synthesis gene in the prefrontal cortex of subjects with schizophrenia.

Authors:  Amanda C Mitchell; Yan Jiang; Cyril Peter; Schahram Akbarian
Journal:  Schizophr Res       Date:  2014-10-31       Impact factor: 4.939

4.  Regenerative medicine: targeted genome editing in vivo.

Authors:  Lixia Wang; Jun Wu; Weiwei Fang; Guang-Hui Liu; Juan Carlos Izpisua Belmonte
Journal:  Cell Res       Date:  2015-01-30       Impact factor: 25.617

5.  Come together, right now….

Authors:  Herman Yeger
Journal:  J Cell Commun Signal       Date:  2015-07-25       Impact factor: 5.782

Review 6.  Induced Pluripotent Stem Cell Models to Enable In Vitro Models for Screening in the Central Nervous System.

Authors:  Joshua G Hunsberger; Anastasia G Efthymiou; Nasir Malik; Mamta Behl; Ivy L Mead; Xianmin Zeng; Anton Simeonov; Mahendra Rao
Journal:  Stem Cells Dev       Date:  2015-04-20       Impact factor: 3.272

Review 7.  Concise review: making and using clinically compliant pluripotent stem cell lines.

Authors:  Melissa K Carpenter; Mahendra S Rao
Journal:  Stem Cells Transl Med       Date:  2015-02-26       Impact factor: 6.940

Review 8.  Genetic predispositions of Parkinson's disease revealed in patient-derived brain cells.

Authors:  Jenne Tran; Helena Anastacio; Cedric Bardy
Journal:  NPJ Parkinsons Dis       Date:  2020-04-24

Review 9.  Current Bioengineering and Regenerative Strategies for the Generation of Kidney Grafts on Demand.

Authors:  Ximo García-Domínguez; Jose S Vicente; Cesar D Vera-Donoso; Francisco Marco-Jimenez
Journal:  Curr Urol Rep       Date:  2017-01       Impact factor: 3.092

Review 10.  Usher syndrome: Hearing loss, retinal degeneration and associated abnormalities.

Authors:  Pranav Mathur; Jun Yang
Journal:  Biochim Biophys Acta       Date:  2014-12-04
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