Literature DB >> 29191331

Genome Editing: The Recent History and Perspective in Cardiovascular Diseases.

Kiran Musunuru1.   

Abstract

The genome-editing field has advanced to a remarkable degree in the last 5 years, culminating in the successful correction of a cardiomyopathy gene mutation in viable human embryos. In this review, the author discusses the basic principles of genome editing, recent advances in clustered regularly interspaced short palindromic repeats and clustered regularly interspaced short palindromic repeats-associated 9 technology, the impact on cardiovascular basic science research, possible therapeutic applications in patients with cardiovascular diseases, and finally the implications of potential clinical uses of human germline genome editing.
Copyright © 2017 American College of Cardiology Foundation. Published by Elsevier Inc. All rights reserved.

Entities:  

Keywords:  CRISPR; Cas9; gene therapy; genetics; genomics

Mesh:

Year:  2017        PMID: 29191331      PMCID: PMC5742864          DOI: 10.1016/j.jacc.2017.10.002

Source DB:  PubMed          Journal:  J Am Coll Cardiol        ISSN: 0735-1097            Impact factor:   24.094


  113 in total

Review 1.  Regulation of DNA repair throughout the cell cycle.

Authors:  Dana Branzei; Marco Foiani
Journal:  Nat Rev Mol Cell Biol       Date:  2008-02-20       Impact factor: 94.444

2.  Correction of a pathogenic gene mutation in human embryos.

Authors:  Hong Ma; Nuria Marti-Gutierrez; Sang-Wook Park; Jun Wu; Yeonmi Lee; Keiichiro Suzuki; Amy Koski; Dongmei Ji; Tomonari Hayama; Riffat Ahmed; Hayley Darby; Crystal Van Dyken; Ying Li; Eunju Kang; A-Reum Park; Daesik Kim; Sang-Tae Kim; Jianhui Gong; Ying Gu; Xun Xu; David Battaglia; Sacha A Krieg; David M Lee; Diana H Wu; Don P Wolf; Stephen B Heitner; Juan Carlos Izpisua Belmonte; Paula Amato; Jin-Soo Kim; Sanjiv Kaul; Shoukhrat Mitalipov
Journal:  Nature       Date:  2017-08-02       Impact factor: 49.962

3.  Genome-wide target specificities of CRISPR RNA-guided programmable deaminases.

Authors:  Daesik Kim; Kayeong Lim; Sang-Tae Kim; Sun-Heui Yoon; Kyoungmi Kim; Seuk-Min Ryu; Jin-Soo Kim
Journal:  Nat Biotechnol       Date:  2017-04-10       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.  Targeted AID-mediated mutagenesis (TAM) enables efficient genomic diversification in mammalian cells.

Authors:  Yunqing Ma; Jiayuan Zhang; Weijie Yin; Zhenchao Zhang; Yan Song; Xing Chang
Journal:  Nat Methods       Date:  2016-10-10       Impact factor: 28.547

6.  Targeted nucleotide editing using hybrid prokaryotic and vertebrate adaptive immune systems.

Authors:  Keiji Nishida; Takayuki Arazoe; Nozomu Yachie; Satomi Banno; Mika Kakimoto; Mayura Tabata; Masao Mochizuki; Aya Miyabe; Michihiro Araki; Kiyotaka Y Hara; Zenpei Shimatani; Akihiko Kondo
Journal:  Science       Date:  2016-08-04       Impact factor: 47.728

7.  Safety and efficacy of RNAi therapy for transthyretin amyloidosis.

Authors:  Teresa Coelho; David Adams; Ana Silva; Pierre Lozeron; Philip N Hawkins; Timothy Mant; Javier Perez; Joseph Chiesa; Steve Warrington; Elizabeth Tranter; Malathy Munisamy; Rick Falzone; Jamie Harrop; Jeffrey Cehelsky; Brian R Bettencourt; Mary Geissler; James S Butler; Alfica Sehgal; Rachel E Meyers; Qingmin Chen; Todd Borland; Renta M Hutabarat; Valerie A Clausen; Rene Alvarez; Kevin Fitzgerald; Christina Gamba-Vitalo; Saraswathy V Nochur; Akshay K Vaishnaw; Dinah W Y Sah; Jared A Gollob; Ole B Suhr
Journal:  N Engl J Med       Date:  2013-08-29       Impact factor: 91.245

Review 8.  A Comprehensive TALEN-Based Knockout Library for Generating Human-Induced Pluripotent Stem Cell-Based Models for Cardiovascular Diseases.

Authors:  Ioannis Karakikes; Vittavat Termglinchan; Diana A Cepeda; Jaecheol Lee; Sebastian Diecke; Ayal Hendel; Ilanit Itzhaki; Mohamed Ameen; Rajani Shrestha; Haodi Wu; Ning Ma; Ning-Yi Shao; Timon Seeger; Nicole Woo; Kitchener D Wilson; Elena Matsa; Matthew H Porteus; Vittorio Sebastiano; Joseph C Wu
Journal:  Circ Res       Date:  2017-02-28       Impact factor: 17.367

9.  Recessive cardiac phenotypes in induced pluripotent stem cell models of Jervell and Lange-Nielsen syndrome: disease mechanisms and pharmacological rescue.

Authors:  Miao Zhang; Cristina D'Aniello; Arie O Verkerk; Eva Wrobel; Stefan Frank; Dorien Ward-van Oostwaard; Ilaria Piccini; Christian Freund; Jyoti Rao; Guiscard Seebohm; Douwe E Atsma; Eric Schulze-Bahr; Christine L Mummery; Boris Greber; Milena Bellin
Journal:  Proc Natl Acad Sci U S A       Date:  2014-12-01       Impact factor: 11.205

10.  iPSC-derived cardiomyocytes reveal abnormal TGF-β signalling in left ventricular non-compaction cardiomyopathy.

Authors:  Kazuki Kodo; Sang-Ging Ong; Fereshteh Jahanbani; Vittavat Termglinchan; Keiichi Hirono; Kolsoum InanlooRahatloo; Antje D Ebert; Praveen Shukla; Oscar J Abilez; Jared M Churko; Ioannis Karakikes; Gwanghyun Jung; Fukiko Ichida; Sean M Wu; Michael P Snyder; Daniel Bernstein; Joseph C Wu
Journal:  Nat Cell Biol       Date:  2016-09-19       Impact factor: 28.824

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  4 in total

Review 1.  Multigene CRISPR/Cas9 genome editing of hybrid proline rich proteins (HyPRPs) for sustainable multi-stress tolerance in crops: the review of a promising approach.

Authors:  Banashree Saikia; Sanjay Singh; Johni Debbarma; Natarajan Velmurugan; Hariprasanna Dekaboruah; Kallare P Arunkumar; Channakeshavaiah Chikkaputtaiah
Journal:  Physiol Mol Biol Plants       Date:  2020-04-20

Review 2.  Angiopoietin-Like 3: From Discovery to Therapeutic Gene Editing.

Authors:  Xiao Wang; Kiran Musunuru
Journal:  JACC Basic Transl Sci       Date:  2019-10-28

Review 3.  Gene surgery: Potential applications for human diseases.

Authors:  Ayman El-Kenawy; Bachir Benarba; Adriana Freitas Neves; Thaise Gonçalves de Araujo; Bee Ling Tan; Adel Gouri
Journal:  EXCLI J       Date:  2019-10-11       Impact factor: 4.068

Review 4.  Moving toward genome-editing therapies for cardiovascular diseases.

Authors:  Kiran Musunuru
Journal:  J Clin Invest       Date:  2022-01-04       Impact factor: 14.808

  4 in total

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