Stem Cell Reports Resource Precise Correction of the Dystrophin Gene in DuchenneMuscular Dystrophy Patient Induced Pluripotent Stem Cells by TALEN and CRISPR-Cas9
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چکیده
Hongmei Lisa Li,1 Naoko Fujimoto,1,2 Noriko Sasakawa,1 Saya Shirai,1 Tokiko Ohkame,1 Tetsushi Sakuma,4 Michihiro Tanaka,1 Naoki Amano,1 Akira Watanabe,1 Hidetoshi Sakurai,1 Takashi Yamamoto,4 Shinya Yamanaka,1,2,5 and Akitsu Hotta1,2,3,* 1Center for iPS Cell Research and Application, Kyoto University, Kyoto 606-8507, Japan 2iCeMS, Kyoto University, Kyoto 606-8501, Japan 3PRESTO, Japan Science and Technology Agency, Kawaguchi 332-0012, Japan 4Department of Mathematical and Life Sciences, Graduate School of Science, Hiroshima University, Higashi-Hiroshima 739-8526, Japan 5Gladstone Institute of Cardiovascular Disease, San Francisco, CA 94158, USA *Correspondence: [email protected] http://dx.doi.org/10.1016/j.stemcr.2014.10.013 This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/3.0/).
منابع مشابه
Precise Correction of the Dystrophin Gene in Duchenne Muscular Dystrophy Patient Induced Pluripotent Stem Cells by TALEN and CRISPR-Cas9
Duchenne muscular dystrophy (DMD) is a severe muscle-degenerative disease caused by a mutation in the dystrophin gene. Genetic correction of patient-derived induced pluripotent stem cells (iPSCs) by TALENs or CRISPR-Cas9 holds promise for DMD gene therapy; however, the safety of such nuclease treatment must be determined. Using a unique k-mer database, we systematically identified a unique targ...
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تاریخ انتشار 2015