First test of a "high-density injection" protocol for myogenic cell transplantation throughout large volumes of muscles in a Duchenne muscular dystrophy patient:: eighteen months follow-up
First test of a "high-density injection" protocol for myogenic cell transplantation throughout large volumes of muscles in a Duchenne muscular dystrophy patient:: eighteen months follow-up
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DOI：
10.1016/j.nmd.2006.10.003
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发表时间：
2007-01-01
影响因子：
2.8
通讯作者：
Tremblay, Jacques P.
中科院分区：
文献类型：
--
作者：
Skuk, Daniel;Goulet, Marlyne;Tremblay, Jacques P.
一位26岁的杜氏肌营养不良（DMD）患者接受了正常的肌肉前体细胞，在体外增殖，并将其植入鱼际隆起、肱二头肌和腓肠肌的一部分，注射间距为1mm或更小。对侧腓肠肌注射生理盐水。他克莫司对患者免疫抑制。细胞移植方案耐受性良好，不会引起永久性后遗症。部分注射部位于移植后1、14、18个月行活检。肌肉被脂肪和纤维化所取代。在腓肠肌细胞移植部位，27.5%的肌纤维在移植后1个月表达供体来源的肌营养不良蛋白，34.5%在移植后18个月表达供体来源的肌营养不良蛋白。对侧腓肠肌肌营养不良蛋白阴性。在肱二头肌中几乎没有肌纤维，只观察到两条肌营养不良蛋白阳性的肌纤维。综上所述，“高密度注射”方案对于DMD患者的肌内细胞移植是可行的，并观察到供体来源的肌营养不良蛋白的长期表达。(C) 2006 Elsevier B.V.版权所有
A 26-years old Duchenne muscular dystrophy (DMD) patient received normal muscle-precursor cells, proliferated in vitro and implanted in a thenar eminence, biceps brachii, and in a portion of a gastrocnemius by injections placed 1 mm from each other or less. Saline was injected in the contralateral gastrocnemius. The patient was immunosuppressed with tacrolimus. The protocol of cell transplantation was well tolerated and did not cause permanent sequels. Some injected sites were biopsied at 1, 14 and 18 months post-transplantation. Muscles were replaced by fat and fibrosis. In the cell-grafted site of the gastrocnemius, 27.5% of the myofiber profiles expressed donor-derived dystrophin 1 month post-transplantation and 34.5% 18 months post-transplantation. The contralateral gastrocnemius was dystrophin-negative. Myofibers were virtually absent in the biceps brachii, where only two dystrophin-positive myofibers were observed. In conclusion, a "high-density injection" protocol was feasible for intramuscular cell-transplantation in a DMD patient and long-term expression of donor-derived dystrophin was observed. (C) 2006 Elsevier B.V. All rights reserved.