Long-Term Follow-Up of the Motor and Cardiopulmonary Function in A DMD Patient after Bone Marrow Tra

来源 :The 12th Annual Asian and Oceanian Myology Center (AOMC)Scie | 被引量 : 0次 | 上传用户:lxlove584
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Objective:Duchenne muscular dystrophy (DMD) is a severe progressive muscular disorder caused by mutations in thedystrophingene.Stem cells transplanted into lethally irradiatedmdxmice and double knock out (DKO) mice can produce normal myofibers and improve the physical activities of the mice.Clinical studies for DMD patients also showed donor-derived myofibers on the recipients muscle, dystrophin expression and no clinical deterioration.We present a DMD patient who underwent bone marrow stem cell transplantation and had periodic follow-up for over 7 years.
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