Literature DB >> 27916992

Gene Editing for Duchenne Muscular Dystrophy Using the CRISPR/Cas9 Technology: The Importance of Fine-tuning the Approach.

Jacques P Tremblay1, Jean-Paul Iyombe-Engembe2, Benjamin Duchêne2, Dominique L Ouellet2.   

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Year:  2016        PMID： 27916992      PMCID： PMC5154487          DOI： 10.1038/mt.2016.191

Source DB:  PubMed          Journal:  Mol Ther        ISSN： 1525-0016            Impact factor:   11.454


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  14 in total

1.  CRISPR/Cas9 Flexes Its Muscles: In Vivo Somatic Gene Editing for Muscular Dystrophy.

Authors:  Thierry VandenDriessche; Marinee K Chuah
Journal:  Mol Ther       Date:  2016-03       Impact factor: 11.454

2.  Becker muscular dystrophy severity is linked to the structure of dystrophin.

Authors:  Aurélie Nicolas; Céline Raguénès-Nicol; Rabah Ben Yaou; Sarah Ameziane-Le Hir; Angélique Chéron; Véronique Vié; Mireille Claustres; France Leturcq; Olivier Delalande; Jean-François Hubert; Sylvie Tuffery-Giraud; Emmanuel Giudice; Elisabeth Le Rumeur
Journal:  Hum Mol Genet       Date:  2014-10-27       Impact factor: 6.150

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.  In vivo gene editing in dystrophic mouse muscle and muscle stem cells.

Authors:  Mohammadsharif Tabebordbar; Kexian Zhu; Jason K W Cheng; Wei Leong Chew; Jeffrey J Widrick; Winston X Yan; Claire Maesner; Elizabeth Y Wu; Ru Xiao; F Ann Ran; Le Cong; Feng Zhang; Luk H Vandenberghe; George M Church; Amy J Wagers
Journal:  Science       Date:  2015-12-31       Impact factor: 47.728

Review 6.  Overview on DMD exon skipping.

Authors:  Annemieke Aartsma-Rus
Journal:  Methods Mol Biol       Date:  2012

7.  CRISPR-mediated Genome Editing Restores Dystrophin Expression and Function in mdx Mice.

Authors:  Li Xu; Ki Ho Park; Lixia Zhao; Jing Xu; Mona El Refaey; Yandi Gao; Hua Zhu; Jianjie Ma; Renzhi Han
Journal:  Mol Ther       Date:  2015-10-09       Impact factor: 11.454

8.  Generation and characterization of transgenic mice with the full-length human DMD gene.

Authors:  Peter A C 't Hoen; Emile J de Meijer; Judith M Boer; Rolf H A M Vossen; Rolf Turk; Ronald G H J Maatman; Kay E Davies; Gert-Jan B van Ommen; Judith C T van Deutekom; Johan T den Dunnen
Journal:  J Biol Chem       Date:  2007-12-13       Impact factor: 5.157

9.  The TREAT-NMD DMD Global Database: analysis of more than 7,000 Duchenne muscular dystrophy mutations.

Authors:  Catherine L Bladen; David Salgado; Soledad Monges; Maria E Foncuberta; Kyriaki Kekou; Konstantina Kosma; Hugh Dawkins; Leanne Lamont; Anna J Roy; Teodora Chamova; Velina Guergueltcheva; Sophelia Chan; Lawrence Korngut; Craig Campbell; Yi Dai; Jen Wang; Nina Barišić; Petr Brabec; Jaana Lahdetie; Maggie C Walter; Olivia Schreiber-Katz; Veronika Karcagi; Marta Garami; Venkatarman Viswanathan; Farhad Bayat; Filippo Buccella; En Kimura; Zaïda Koeks; Janneke C van den Bergen; Miriam Rodrigues; Richard Roxburgh; Anna Lusakowska; Anna Kostera-Pruszczyk; Janusz Zimowski; Rosário Santos; Elena Neagu; Svetlana Artemieva; Vedrana Milic Rasic; Dina Vojinovic; Manuel Posada; Clemens Bloetzer; Pierre-Yves Jeannet; Franziska Joncourt; Jordi Díaz-Manera; Eduard Gallardo; A Ayşe Karaduman; Haluk Topaloğlu; Rasha El Sherif; Angela Stringer; Andriy V Shatillo; Ann S Martin; Holly L Peay; Matthew I Bellgard; Jan Kirschner; Kevin M Flanigan; Volker Straub; Kate Bushby; Jan Verschuuren; Annemieke Aartsma-Rus; Christophe Béroud; Hanns Lochmüller
Journal:  Hum Mutat       Date:  2015-03-17       Impact factor: 4.878

Review 10.  Current understanding of molecular pathology and treatment of cardiomyopathy in duchenne muscular dystrophy.

Authors:  Tirsa L E van Westering; Corinne A Betts; Matthew J A Wood
Journal:  Molecules       Date:  2015-05-15       Impact factor: 4.411
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  4 in total

1.  AAV9 Edits Muscle Stem Cells in Normal and Dystrophic Adult Mice.

Authors:  Michael E Nance; Ruicheng Shi; Chady H Hakim; Nalinda B Wasala; Yongping Yue; Xiufang Pan; Tracy Zhang; Carolyn A Robinson; Sean X Duan; Gang Yao; N Nora Yang; Shi-Jie Chen; Kathryn R Wagner; Charles A Gersbach; Dongsheng Duan
Journal:  Mol Ther       Date:  2019-07-03       Impact factor: 11.454

2.  Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy.

Authors:  Leonela Amoasii; John C W Hildyard; Hui Li; Efrain Sanchez-Ortiz; Alex Mireault; Daniel Caballero; Rachel Harron; Thaleia-Rengina Stathopoulou; Claire Massey; John M Shelton; Rhonda Bassel-Duby; Richard J Piercy; Eric N Olson
Journal:  Science       Date:  2018-08-30       Impact factor: 47.728

Review 3.  Genome editing technologies: CRISPR, LEAPER, RESTORE, ARCUT, SATI, and RESCUE.

Authors:  Senay Görücü Yilmaz
Journal:  EXCLI J       Date:  2021-01-04       Impact factor: 4.068

4.  In vivo non-invasive monitoring of dystrophin correction in a new Duchenne muscular dystrophy reporter mouse.

Authors:  Leonela Amoasii; Hui Li; Yu Zhang; Yi-Li Min; Efrain Sanchez-Ortiz; John M Shelton; Chengzu Long; Alex A Mireault; Samadrita Bhattacharyya; John R McAnally; Rhonda Bassel-Duby; Eric N Olson
Journal:  Nat Commun       Date:  2019-10-04       Impact factor: 14.919
  4 in total

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