Literature DB >> 27218233

Genome engineering tools for building cellular models of disease.

Jennie Lin1, Kiran Musunuru2,3.   

Abstract

With the recent development of methods for genome editing of human pluripotent stem cells, study of the genetic basis of human diseases has been rapidly advancing. Genome-edited differentiated stem cells have provided new and more accurate insights into genomic underpinnings of diseases for which there have not been adequate treatments, and moving toward clinical application of genome editing holds great promise for acceleration of therapeutic translation. Here, we review recent advances in genome-editing technologies and their application to human biology in disease modeling and beyond.
© 2016 Federation of European Biochemical Societies.

Entities:  

Keywords:  CRISPR/Cas9; TALENs; genome editing; induced pluripotent stem cell; zinc finger nucleases

Mesh：

Substances：

Year:  2016        PMID： 27218233      PMCID： PMC5881911          DOI： 10.1111/febs.13763

Source DB:  PubMed          Journal:  FEBS J        ISSN： 1742-464X            Impact factor:   5.542


  76 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.  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

4.  In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy.

Authors:  Christopher E Nelson; Chady H Hakim; David G Ousterout; Pratiksha I Thakore; Eirik A Moreb; Ruth M Castellanos Rivera; Sarina Madhavan; Xiufang Pan; F Ann Ran; Winston X Yan; Aravind Asokan; Feng Zhang; Dongsheng Duan; Charles A Gersbach
Journal:  Science       Date:  2015-12-31       Impact factor: 47.728

5.  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

6.  Human neural crest cells contribute to coat pigmentation in interspecies chimeras after in utero injection into mouse embryos.

Authors:  Malkiel A Cohen; Katherine J Wert; Johanna Goldmann; Styliani Markoulaki; Yosef Buganim; Dongdong Fu; Rudolf Jaenisch
Journal:  Proc Natl Acad Sci U S A       Date:  2016-01-25       Impact factor: 11.205

7.  Oligomerized pool engineering (OPEN): an 'open-source' protocol for making customized zinc-finger arrays.

Authors:  Morgan L Maeder; Stacey Thibodeau-Beganny; Jeffry D Sander; Daniel F Voytas; J Keith Joung
Journal:  Nat Protoc       Date:  2009-09-17       Impact factor: 13.491

8.  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

9.  CAS9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering.

Authors:  Prashant Mali; John Aach; P Benjamin Stranges; Kevin M Esvelt; Mark Moosburner; Sriram Kosuri; Luhan Yang; George M Church
Journal:  Nat Biotechnol       Date:  2013-08-01       Impact factor: 54.908

10.  Common variants associated with plasma triglycerides and risk for coronary artery disease.

Authors:  Ron Do; Cristen J Willer; Ellen M Schmidt; Sebanti Sengupta; Chi Gao; Gina M Peloso; Stefan Gustafsson; Stavroula Kanoni; Andrea Ganna; Jin Chen; Martin L Buchkovich; Samia Mora; Jacques S Beckmann; Jennifer L Bragg-Gresham; Hsing-Yi Chang; Ayşe Demirkan; Heleen M Den Hertog; Louise A Donnelly; Georg B Ehret; Tõnu Esko; Mary F Feitosa; Teresa Ferreira; Krista Fischer; Pierre Fontanillas; Ross M Fraser; Daniel F Freitag; Deepti Gurdasani; Kauko Heikkilä; Elina Hyppönen; Aaron Isaacs; Anne U Jackson; Asa Johansson; Toby Johnson; Marika Kaakinen; Johannes Kettunen; Marcus E Kleber; Xiaohui Li; Jian'an Luan; Leo-Pekka Lyytikäinen; Patrik K E Magnusson; Massimo Mangino; Evelin Mihailov; May E Montasser; Martina Müller-Nurasyid; Ilja M Nolte; Jeffrey R O'Connell; Cameron D Palmer; Markus Perola; Ann-Kristin Petersen; Serena Sanna; Richa Saxena; Susan K Service; Sonia Shah; Dmitry Shungin; Carlo Sidore; Ci Song; Rona J Strawbridge; Ida Surakka; Toshiko Tanaka; Tanya M Teslovich; Gudmar Thorleifsson; Evita G Van den Herik; Benjamin F Voight; Kelly A Volcik; Lindsay L Waite; Andrew Wong; Ying Wu; Weihua Zhang; Devin Absher; Gershim Asiki; Inês Barroso; Latonya F Been; Jennifer L Bolton; Lori L Bonnycastle; Paolo Brambilla; Mary S Burnett; Giancarlo Cesana; Maria Dimitriou; Alex S F Doney; Angela Döring; Paul Elliott; Stephen E Epstein; Gudmundur Ingi Eyjolfsson; Bruna Gigante; Mark O Goodarzi; Harald Grallert; Martha L Gravito; Christopher J Groves; Göran Hallmans; Anna-Liisa Hartikainen; Caroline Hayward; Dena Hernandez; Andrew A Hicks; Hilma Holm; Yi-Jen Hung; Thomas Illig; Michelle R Jones; Pontiano Kaleebu; John J P Kastelein; Kay-Tee Khaw; Eric Kim; Norman Klopp; Pirjo Komulainen; Meena Kumari; Claudia Langenberg; Terho Lehtimäki; Shih-Yi Lin; Jaana Lindström; Ruth J F Loos; François Mach; Wendy L McArdle; Christa Meisinger; Braxton D Mitchell; Gabrielle Müller; Ramaiah Nagaraja; Narisu Narisu; Tuomo V M Nieminen; Rebecca N Nsubuga; Isleifur Olafsson; Ken K Ong; Aarno Palotie; Theodore Papamarkou; Cristina Pomilla; Anneli Pouta; Daniel J Rader; Muredach P Reilly; Paul M Ridker; Fernando Rivadeneira; Igor Rudan; Aimo Ruokonen; Nilesh Samani; Hubert Scharnagl; Janet Seeley; Kaisa Silander; Alena Stančáková; Kathleen Stirrups; Amy J Swift; Laurence Tiret; Andre G Uitterlinden; L Joost van Pelt; Sailaja Vedantam; Nicholas Wainwright; Cisca Wijmenga; Sarah H Wild; Gonneke Willemsen; Tom Wilsgaard; James F Wilson; Elizabeth H Young; Jing Hua Zhao; Linda S Adair; Dominique Arveiler; Themistocles L Assimes; Stefania Bandinelli; Franklyn Bennett; Murielle Bochud; Bernhard O Boehm; Dorret I Boomsma; Ingrid B Borecki; Stefan R Bornstein; Pascal Bovet; Michel Burnier; Harry Campbell; Aravinda Chakravarti; John C Chambers; Yii-Der Ida Chen; Francis S Collins; Richard S Cooper; John Danesh; George Dedoussis; Ulf de Faire; Alan B Feranil; Jean Ferrières; Luigi Ferrucci; Nelson B Freimer; Christian Gieger; Leif C Groop; Vilmundur Gudnason; Ulf Gyllensten; Anders Hamsten; Tamara B Harris; Aroon Hingorani; Joel N Hirschhorn; Albert Hofman; G Kees Hovingh; Chao Agnes Hsiung; Steve E Humphries; Steven C Hunt; Kristian Hveem; Carlos Iribarren; Marjo-Riitta Järvelin; Antti Jula; Mika Kähönen; Jaakko Kaprio; Antero Kesäniemi; Mika Kivimaki; Jaspal S Kooner; Peter J Koudstaal; Ronald M Krauss; Diana Kuh; Johanna Kuusisto; Kirsten O Kyvik; Markku Laakso; Timo A Lakka; Lars Lind; Cecilia M Lindgren; Nicholas G Martin; Winfried März; Mark I McCarthy; Colin A McKenzie; Pierre Meneton; Andres Metspalu; Leena Moilanen; Andrew D Morris; Patricia B Munroe; Inger Njølstad; Nancy L Pedersen; Chris Power; Peter P Pramstaller; Jackie F Price; Bruce M Psaty; Thomas Quertermous; Rainer Rauramaa; Danish Saleheen; Veikko Salomaa; Dharambir K Sanghera; Jouko Saramies; Peter E H Schwarz; Wayne H-H Sheu; Alan R Shuldiner; Agneta Siegbahn; Tim D Spector; Kari Stefansson; David P Strachan; Bamidele O Tayo; Elena Tremoli; Jaakko Tuomilehto; Matti Uusitupa; Cornelia M van Duijn; Peter Vollenweider; Lars Wallentin; Nicholas J Wareham; John B Whitfield; Bruce H R Wolffenbuttel; David Altshuler; Jose M Ordovas; Eric Boerwinkle; Colin N A Palmer; Unnur Thorsteinsdottir; Daniel I Chasman; Jerome I Rotter; Paul W Franks; Samuli Ripatti; L Adrienne Cupples; Manjinder S Sandhu; Stephen S Rich; Michael Boehnke; Panos Deloukas; Karen L Mohlke; Erik Ingelsson; Goncalo R Abecasis; Mark J Daly; Benjamin M Neale; Sekar Kathiresan
Journal:  Nat Genet       Date:  2013-10-06       Impact factor: 38.330
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  11 in total

1.  Using zebrafish to study skeletal genomics.

Authors:  Ronald Y Kwon; Claire J Watson; David Karasik
Journal:  Bone       Date:  2019-02-11       Impact factor: 4.398

2.  Complexities of Understanding Function from CKD-Associated DNA Variants.

Authors:  Jennie Lin; Katalin Susztak
Journal:  Clin J Am Soc Nephrol       Date:  2020-06-08       Impact factor: 8.237

3.  From Genotype to Phenotype: A Primer on the Functional Follow-up of Genome-Wide Association Studies in Cardiovascular Disease.

Authors:  Jennie Lin; Kiran Musunuru
Journal:  Circ Genom Precis Med       Date:  2018-01-30

4.  Targeted mutagenesis in human iPSCs using CRISPR genome-editing tools.

Authors:  Yicheng Long; Thomas R Cech
Journal:  Methods       Date:  2021-01-12       Impact factor: 4.647

Review 5.  May I Cut in? Gene Editing Approaches in Human Induced Pluripotent Stem Cells.

Authors:  Nicholas Brookhouser; Sreedevi Raman; Christopher Potts; David A Brafman
Journal:  Cells       Date:  2017-02-06       Impact factor: 6.600

6.  CIRCLE-seq: a highly sensitive in vitro screen for genome-wide CRISPR-Cas9 nuclease off-targets.

Authors:  Shengdar Q Tsai; Nhu T Nguyen; Jose Malagon-Lopez; Ved V Topkar; Martin J Aryee; J Keith Joung
Journal:  Nat Methods       Date:  2017-05-01       Impact factor: 28.547

7.  Highly efficient genome editing for single-base substitutions using optimized ssODNs with Cas9-RNPs.

Authors:  Sachiko Okamoto; Yasunori Amaishi; Izumi Maki; Tatsuji Enoki; Junichi Mineno
Journal:  Sci Rep       Date:  2019-03-18       Impact factor: 4.379

8.  CRISPR/Cas9-mediated one step bi-allelic change of genomic DNA in iPSCs and human RPE cells in vitro with dual antibiotic selection.

Authors:  Wasu Supharattanasitthi; Emil Carlsson; Umar Sharif; Luminita Paraoan
Journal:  Sci Rep       Date:  2019-01-17       Impact factor: 4.379

Review 9.  Using induced pluripotent stem cells for modeling Parkinson's disease.

Authors:  Minjing Ke; Cheong-Meng Chong; Huanxing Su
Journal:  World J Stem Cells       Date:  2019-09-26       Impact factor: 5.326

10.  Profiling APOL1 Nephropathy Risk Variants in Genome-Edited Kidney Organoids with Single-Cell Transcriptomics.

Authors:  Esther Liu; Behram Radmanesh; Byungha H Chung; Benjamin S Freedman; Jennie Lin; Michael D Donnan; Dan Yi; Amal Dadi; Kelly D Smith; Jonathan Himmelfarb; Mingyao Li
Journal:  Kidney360       Date:  2020-03
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