Article ID | Journal | Published Year | Pages | File Type |
---|---|---|---|---|
8425621 | Stem Cell Research | 2018 | 4 Pages |
Abstract
Duchenne muscular dystrophy (DMD) is an X-linked disease caused by mutations in the DMD gene, which spans ~Â 2.4Â Mb of genomic sequence at locus Xp21. This mutation results in the loss of the protein dystrophin. DMD patients die in their second or third decade due to either respiratory failure or cardiomyopathy, as the absence of dystrophin leads to myofiber membrane fragility and necrosis, eventually resulting in muscle atrophy and contractures. Currently, there is no effective treatment for DMD, therefore induced pluripotent stem cells from DMD patients would be a powerful tool for studying disease mechanisms.
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Authors
Xie Yuhuan, Xie Yingjun, Xue Yanting, Chen Yuchang, Song Bing, Li Shaoying, Li Haoxian, Xian Yexing, Ouyang Shuming, Xiong Zeyu, Sun Xiaofang,