Literature DB >> 26748756

Genome Editing in Human Pluripotent Stem Cells: Approaches, Pitfalls, and Solutions.

William T Hendriks1, Curtis R Warren2, Chad A Cowan3.   

Abstract

Human pluripotent stem cells (hPSCs) with knockout or mutant alleles can be generated using custom-engineered nucleases. Transcription activator-like effector nucleases (TALENs) and clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 nucleases are the most commonly employed technologies for editing hPSC genomes. In this Protocol Review, we provide a brief overview of custom-engineered nucleases in the context of gene editing in hPSCs with a focus on the application of TALENs and CRISPR/Cas9. We will highlight the advantages and disadvantages of each method and discuss theoretical and technical considerations for experimental design.
Copyright © 2016 Elsevier Inc. All rights reserved.

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Year:  2016        PMID： 26748756      PMCID： PMC4709030          DOI： 10.1016/j.stem.2015.12.002

Source DB:  PubMed          Journal:  Cell Stem Cell        ISSN： 1875-9777            Impact factor:   24.633


  111 in total

1.  Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificity.

Authors:  F Ann Ran; Patrick D Hsu; Chie-Yu Lin; Jonathan S Gootenberg; Silvana Konermann; Alexandro E Trevino; David A Scott; Azusa Inoue; Shogo Matoba; Yi Zhang; Feng Zhang
Journal:  Cell       Date:  2013-08-29       Impact factor: 41.582

2.  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

3.  A method for genetic modification of human embryonic stem cells using electroporation.

Authors:  Magdaline Costa; Mirella Dottori; Koula Sourris; Pegah Jamshidi; Tanya Hatzistavrou; Richard Davis; Lisa Azzola; Steven Jackson; Sue Mei Lim; Martin Pera; Andrew G Elefanty; Edouard G Stanley
Journal:  Nat Protoc       Date:  2007       Impact factor: 13.491

Review 4.  Repair of strand breaks by homologous recombination.

Authors:  Maria Jasin; Rodney Rothstein
Journal:  Cold Spring Harb Perspect Biol       Date:  2013-11-01       Impact factor: 10.005

5.  Highly efficient endogenous human gene correction using designed zinc-finger nucleases.

Authors:  Fyodor D Urnov; Jeffrey C Miller; Ya-Li Lee; Christian M Beausejour; Jeremy M Rock; Sheldon Augustus; Andrew C Jamieson; Matthew H Porteus; Philip D Gregory; Michael C Holmes
Journal:  Nature       Date:  2005-04-03       Impact factor: 49.962

6.  TALEN-based gene correction for epidermolysis bullosa.

Authors:  Mark J Osborn; Colby G Starker; Amber N McElroy; Beau R Webber; Megan J Riddle; Lily Xia; Anthony P DeFeo; Richard Gabriel; Manfred Schmidt; Christof von Kalle; Daniel F Carlson; Morgan L Maeder; J Keith Joung; John E Wagner; Daniel F Voytas; Bruce R Blazar; Jakub Tolar
Journal:  Mol Ther       Date:  2013-04-02       Impact factor: 11.454

7.  Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells.

Authors:  Ayal Hendel; Rasmus O Bak; Joseph T Clark; Andrew B Kennedy; Daniel E Ryan; Subhadeep Roy; Israel Steinfeld; Benjamin D Lunstad; Robert J Kaiser; Alec B Wilkens; Rosa Bacchetta; Anya Tsalenko; Douglas Dellinger; Laurakay Bruhn; Matthew H Porteus
Journal:  Nat Biotechnol       Date:  2015-06-29       Impact factor: 54.908

8.  Small molecules enhance CRISPR genome editing in pluripotent stem cells.

Authors:  Chen Yu; Yanxia Liu; Tianhua Ma; Kai Liu; Shaohua Xu; Yu Zhang; Honglei Liu; Marie La Russa; Min Xie; Sheng Ding; Lei S Qi
Journal:  Cell Stem Cell       Date:  2015-02-05       Impact factor: 24.633

9.  Stem cells: balancing resistance and sensitivity to DNA damage.

Authors:  Julia C Liu; Paul H Lerou; Galit Lahav
Journal:  Trends Cell Biol       Date:  2014-04-07       Impact factor: 20.808

10.  Enhanced efficiency of human pluripotent stem cell genome editing through replacing TALENs with CRISPRs.

Authors:  Qiurong Ding; Stephanie N Regan; Yulei Xia; Leoníe A Oostrom; Chad A Cowan; Kiran Musunuru
Journal:  Cell Stem Cell       Date:  2013-04-04       Impact factor: 24.633
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  39 in total

Review 1.  iPS cells in the study of PD molecular pathogenesis.

Authors:  Melanie M Cobb; Abinaya Ravisankar; Gaia Skibinski; Steven Finkbeiner
Journal:  Cell Tissue Res       Date:  2017-12-12       Impact factor: 5.249

Review 2.  Applications of genetically engineered human pluripotent stem cell reporters in cardiac stem cell biology.

Authors:  Joe Z Zhang; Hongchao Guo; Joseph C Wu
Journal:  Curr Opin Biotechnol       Date:  2018-03-24       Impact factor: 9.740

Review 3.  Stem cell-derived organoids to model gastrointestinal facets of cystic fibrosis.

Authors:  Meike Hohwieler; Lukas Perkhofer; Stefan Liebau; Thomas Seufferlein; Martin Müller; Anett Illing; Alexander Kleger
Journal:  United European Gastroenterol J       Date:  2016-09-21       Impact factor: 4.623

Review 4.  Adeno-Associated Virus Vectors and Stem Cells: Friends or Foes?

Authors:  Nolan Brown; Liujiang Song; Nageswara R Kollu; Matthew L Hirsch
Journal:  Hum Gene Ther       Date:  2017-06       Impact factor: 5.695

Review 5.  Gene Editing in Human Pluripotent Stem Cells: Recent Advances for Clinical Therapies.

Authors:  Hatice Burcu Şişli; Taha Bartu Hayal; Selin Seçkin; Selinay Şenkal; Binnur Kıratlı; Fikrettin Şahin; Ayşegül Doğan
Journal:  Adv Exp Med Biol       Date:  2020       Impact factor: 2.622

Review 6.  Perspectives and Challenges of Pluripotent Stem Cells in Cardiac Arrhythmia Research.

Authors:  Alexander Goedel; Ilaria My; Daniel Sinnecker; Alessandra Moretti
Journal:  Curr Cardiol Rep       Date:  2017-03       Impact factor: 2.931

7.  Modeling and correction of structural variations in patient-derived iPSCs using CRISPR/Cas9.

Authors:  Chul-Yong Park; Jin Jea Sung; Sang-Hwi Choi; Dongjin R Lee; In-Hyun Park; Dong-Wook Kim
Journal:  Nat Protoc       Date:  2016-10-06       Impact factor: 13.491

Review 8.  Engineering Hematopoietic Stem Cells: Lessons from Development.

Authors:  R Grant Rowe; Joseph Mandelbaum; Leonard I Zon; George Q Daley
Journal:  Cell Stem Cell       Date:  2016-06-02       Impact factor: 24.633

9.  Determining the Pathogenicity of a Genomic Variant of Uncertain Significance Using CRISPR/Cas9 and Human-Induced Pluripotent Stem Cells.

Authors:  Ning Ma; Joe Z Zhang; Ilanit Itzhaki; Sophia L Zhang; Haodong Chen; Francois Haddad; Tomoya Kitani; Kitchener D Wilson; Lei Tian; Rajani Shrestha; Haodi Wu; Chi Keung Lam; Nazish Sayed; Joseph C Wu
Journal:  Circulation       Date:  2018-12-04       Impact factor: 29.690

10.  Comprehensive Protocols for CRISPR/Cas9-based Gene Editing in Human Pluripotent Stem Cells.

Authors:  David P Santos; Evangelos Kiskinis; Kevin Eggan; Florian T Merkle
Journal:  Curr Protoc Stem Cell Biol       Date:  2016-08-17
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