Literature DB >> 26917062

Scalpel or Straitjacket: CRISPR/Cas9 Approaches for Muscular Dystrophies.

Charis L Himeda1, Takako I Jones1, Peter L Jones2.   

Abstract

Versatility of CRISPR/Cas9-based platforms makes them promising tools for the correction of diverse genetic/epigenetic disorders. Here we contrast the use of these genome editing tools in two myopathies with very different molecular origins: Duchenne muscular dystrophy, a monogenetic disease, and facioscapulohumeral muscular dystrophy, an epigenetic disorder with unique therapeutic challenges.
Copyright © 2016 Elsevier Ltd. All rights reserved.

Entities:  

Keywords:  CRISPR; Cas9; DMD; FSHD; genome editing; muscular dystrophy

Mesh：

Substances：

Year:  2016        PMID： 26917062      PMCID： PMC5193161          DOI： 10.1016/j.tips.2016.02.001

Source DB:  PubMed          Journal:  Trends Pharmacol Sci        ISSN： 0165-6147            Impact factor:   14.819


  12 in total

Review 1.  High-Throughput Silencing Using the CRISPR-Cas9 System: A Review of the Benefits and Challenges.

Authors:  Mark Wade
Journal:  J Biomol Screen       Date:  2015-05-22

Review 2.  Facioscapulohumeral muscular dystrophy as a model for epigenetic regulation and disease.

Authors:  Charis L Himeda; Takako I Jones; Peter L Jones
Journal:  Antioxid Redox Signal       Date:  2014-12-04       Impact factor: 8.401

3.  Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy.

Authors:  Chengzu Long; Leonela Amoasii; Alex A Mireault; John R McAnally; Hui Li; Efrain Sanchez-Ortiz; Samadrita Bhattacharyya; John M Shelton; Rhonda Bassel-Duby; Eric N Olson
Journal:  Science       Date:  2015-12-31       Impact factor: 47.728

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.  CRISPR/dCas9-mediated Transcriptional Inhibition Ameliorates the Epigenetic Dysregulation at D4Z4 and Represses DUX4-fl in FSH Muscular Dystrophy.

Authors:  Charis L Himeda; Takako I Jones; Peter L Jones
Journal:  Mol Ther       Date:  2015-11-03       Impact factor: 11.454

6.  Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy.

Authors:  David G Ousterout; Ami M Kabadi; Pratiksha I Thakore; William H Majoros; Timothy E Reddy; Charles A Gersbach
Journal:  Nat Commun       Date:  2015-02-18       Impact factor: 14.919

7.  Trans-spliced Cas9 allows cleavage of HBB and CCR5 genes in human cells using compact expression cassettes.

Authors:  Eli J Fine; Caleb M Appleton; Douglas E White; Matthew T Brown; Harshavardhan Deshmukh; Melissa L Kemp; Gang Bao
Journal:  Sci Rep       Date:  2015-07-01       Impact factor: 4.379

8.  Individual epigenetic status of the pathogenic D4Z4 macrosatellite correlates with disease in facioscapulohumeral muscular dystrophy.

Authors:  Takako I Jones; Oliver D King; Charis L Himeda; Sachiko Homma; Jennifer C J Chen; Mary Lou Beermann; Chi Yan; Charles P Emerson; Jeffrey B Miller; Kathryn R Wagner; Peter L Jones
Journal:  Clin Epigenetics       Date:  2015-03-29       Impact factor: 6.551

9.  In vivo genome editing using Staphylococcus aureus Cas9.

Authors:  F Ann Ran; Le Cong; Winston X Yan; David A Scott; Jonathan S Gootenberg; Andrea J Kriz; Bernd Zetsche; Ophir Shalem; Xuebing Wu; Kira S Makarova; Eugene V Koonin; Phillip A Sharp; Feng Zhang
Journal:  Nature       Date:  2015-04-01       Impact factor: 49.962

Review 10.  Genome-editing Technologies for Gene and Cell Therapy.

Authors:  Morgan L Maeder; Charles A Gersbach
Journal:  Mol Ther       Date:  2016-01-12       Impact factor: 11.454
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  4 in total

Review 1.  FSHD Therapeutic Strategies: What Will It Take to Get to Clinic?

Authors:  Charis L Himeda; Peter L Jones
Journal:  J Pers Med       Date:  2022-05-25

Review 2.  Therapeutic Approaches in Facioscapulohumeral Muscular Dystrophy.

Authors:  Justin Cohen; Alec DeSimone; Monkol Lek; Angela Lek
Journal:  Trends Mol Med       Date:  2020-10-19       Impact factor: 11.951

3.  Antisense Oligonucleotides Used to Target the DUX4 mRNA as Therapeutic Approaches in FaciosScapuloHumeral Muscular Dystrophy (FSHD).

Authors:  Eugénie Ansseau; Céline Vanderplanck; Armelle Wauters; Scott Q Harper; Frédérique Coppée; Alexandra Belayew
Journal:  Genes (Basel)       Date:  2017-03-03       Impact factor: 4.096

4.  Targeted epigenetic repression by CRISPR/dSaCas9 suppresses pathogenic DUX4-fl expression in FSHD.

Authors:  Charis L Himeda; Takako I Jones; Peter L Jones
Journal:  Mol Ther Methods Clin Dev       Date:  2020-12-10       Impact factor: 6.698
  4 in total

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