Literature DB >> 26952918

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

Thierry VandenDriessche1,2, Marinee K Chuah1,2.   

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Year:  2016        PMID： 26952918      PMCID： PMC4786932          DOI： 10.1038/mt.2016.29

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


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  29 in total

1.  Efficacy and safety of adeno-associated viral vectors based on serotype 8 and 9 vs. lentiviral vectors for hemophilia B gene therapy.

Authors:  T Vandendriessche; L Thorrez; A Acosta-Sanchez; I Petrus; L Wang; L Ma; L DE Waele; Y Iwasaki; V Gillijns; J M Wilson; D Collen; M K L Chuah
Journal:  J Thromb Haemost       Date:  2006-09-26       Impact factor: 5.824

2.  Recombinant adeno-associated virus serotype 9 leads to preferential cardiac transduction in vivo.

Authors:  Christina A Pacak; Cathryn S Mah; Bijoy D Thattaliyath; Thomas J Conlon; Melissa A Lewis; Denise E Cloutier; Irene Zolotukhin; Alice F Tarantal; Barry J Byrne
Journal:  Circ Res       Date:  2006-07-27       Impact factor: 17.367

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.  Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA.

Authors:  Chengzu Long; John R McAnally; John M Shelton; Alex A Mireault; Rhonda Bassel-Duby; Eric N Olson
Journal:  Science       Date:  2014-08-14       Impact factor: 47.728

5.  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

Review 6.  Adeno-Associated Virus (AAV) Mediated Dystrophin Gene Transfer Studies and Exon Skipping Strategies for Duchenne Muscular Dystrophy (DMD).

Authors:  Klaudia Kawecka; Michael Theodoulides; Yalin Hasoglu; Susan Jarmin; Hanna Kymalainen; Anita Le-Heron; Linda Popplewell; Alberto Malerba; George Dickson; Takis Athanasopoulos
Journal:  Curr Gene Ther       Date:  2015       Impact factor: 4.391

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.  Precise correction of the dystrophin gene in duchenne muscular dystrophy patient induced pluripotent stem cells by TALEN and CRISPR-Cas9.

Authors:  Hongmei Lisa Li; Naoko Fujimoto; Noriko Sasakawa; Saya Shirai; Tokiko Ohkame; Tetsushi Sakuma; Michihiro Tanaka; Naoki Amano; Akira Watanabe; Hidetoshi Sakurai; Takashi Yamamoto; Shinya Yamanaka; Akitsu Hotta
Journal:  Stem Cell Reports       Date:  2014-11-26       Impact factor: 7.765

9.  piggyBac transposons expressing full-length human dystrophin enable genetic correction of dystrophic mesoangioblasts.

Authors:  Mariana Loperfido; Susan Jarmin; Sumitava Dastidar; Mario Di Matteo; Ilaria Perini; Marc Moore; Nisha Nair; Ermira Samara-Kuko; Takis Athanasopoulos; Francesco Saverio Tedesco; George Dickson; Maurilio Sampaolesi; Thierry VandenDriessche; Marinee K Chuah
Journal:  Nucleic Acids Res       Date:  2015-12-17       Impact factor: 16.971

10.  Selection-free gene repair after adenoviral vector transduction of designer nucleases: rescue of dystrophin synthesis in DMD muscle cell populations.

Authors:  Ignazio Maggio; Luca Stefanucci; Josephine M Janssen; Jin Liu; Xiaoyu Chen; Vincent Mouly; Manuel A F V Gonçalves
Journal:  Nucleic Acids Res       Date:  2016-01-13       Impact factor: 16.971
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  4 in total

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

Authors:  Jacques P Tremblay; Jean-Paul Iyombe-Engembe; Benjamin Duchêne; Dominique L Ouellet
Journal:  Mol Ther       Date:  2016-11       Impact factor: 11.454

Review 2.  Current and Emerging Therapies for Duchenne Muscular Dystrophy.

Authors:  Megan Crone; Jean K Mah
Journal:  Curr Treat Options Neurol       Date:  2018-06-23       Impact factor: 3.598

3.  Efficient CRISPR/Cas9-mediated editing of trinucleotide repeat expansion in myotonic dystrophy patient-derived iPS and myogenic cells.

Authors:  Sumitava Dastidar; Simon Ardui; Kshitiz Singh; Debanjana Majumdar; Nisha Nair; Yanfang Fu; Deepak Reyon; Ermira Samara; Mattia F M Gerli; Arnaud F Klein; Wito De Schrijver; Jaitip Tipanee; Sara Seneca; Warut Tulalamba; Hui Wang; Yoke Chin Chai; Peter In't Veld; Denis Furling; Francesco Saverio Tedesco; Joris R Vermeesch; J Keith Joung; Marinee K Chuah; Thierry VandenDriessche
Journal:  Nucleic Acids Res       Date:  2018-09-19       Impact factor: 16.971

Review 4.  CRISPR Editing in Biological and Biomedical Investigation.

Authors:  Han Zhang; Nami McCarty
Journal:  J Cell Biochem       Date:  2017-05-31       Impact factor: 4.429
  4 in total

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