Tremblay J P, Bouchard J P, Malouin F, Théau D, Cottrell F, Collin H, Rouche A, Gilgenkrantz S, Abbadi N, Tremblay M
Centre de recherche en Neurobiologie, Université Laval, Québec, Canada.
Neuromuscul Disord. 1993 Sep-Nov;3(5-6):583-92. doi: 10.1016/0960-8966(93)90121-y.
Monozygotic twin girls, both carriers of Duchenne muscular dystrophy, only one a severe symptomatic carrier and the other asymptomatic due to opposite lyonization, were studied. Myoblast clones were obtained from a muscle biopsy of the asymptomatic carrier. PCR amplification showed that most (94%) of these clones produced normal dystrophin mRNA. Roughly 704 million myoblasts were produced from 119 clones. These myoblasts were transplanted into the extensor carpi radialis (ECR) and in the biceps of one arm of the manifesting carrier while the other arm acted as the control. The strength of the patient was evaluated in a series of pre- and post-tests and a biopsy was obtained about 1 yr after the transplantation. The myoblast injections produced a significant force gain (12%-31%) in wrist extension but no force gain for elbow flexion. Muscle biopsies on the injected and control muscles obtained 1 yr after the injections showed only a small increase in the number of dystrophin positive fibers and the presence of numerous small type II fibers. The small beneficial effect of this transplantation cannot be attributed to immune problems, the donor and the recipient being identical twins, but may be due to a low level of spontaneous muscle regeneration.
对一对单卵双胞胎女孩进行了研究，她们都是杜氏肌营养不良症的携带者，其中一个是严重症状携带者，另一个由于相反的里昂化作用而无症状。从无症状携带者的肌肉活检中获得了成肌细胞克隆。聚合酶链反应（PCR）扩增显示，这些克隆中的大多数（94%）产生正常的抗肌萎缩蛋白mRNA。从119个克隆中产生了约7亿个成肌细胞。将这些成肌细胞移植到症状明显的携带者一侧手臂的桡侧腕伸肌（ECR）和肱二头肌中，而另一侧手臂作为对照。在一系列移植前和移植后的测试中对患者的力量进行了评估，并在移植后约1年进行了活检。成肌细胞注射使腕部伸展力量显著增加（12%-31%），但肘部屈曲力量没有增加。注射后1年对注射肌肉和对照肌肉进行的活检显示，抗肌萎缩蛋白阳性纤维数量仅略有增加，并且存在大量小型II型纤维。这种移植的微小有益效果不能归因于免疫问题，因为供体和受体是同卵双胞胎，而可能是由于自发肌肉再生水平较低。
