Shishkin S S, Terekhov S M, Krokhina T B, Shakhovskaia N I, Podobedova A N, Linnaia G F, Tarasov V I, Ovchinnikov V I, Krakhmaleva I N, Zakharov S F, Ershova E S, Limborskaia S A, Pogoda T V, Zotikov E A, Kut'ina R M, Tarksh M A, Sukhorukov V S, Gerasimova N L
Research Center for Medical Genetics, Russian Academy of Medical Sciences, Moscow, 115478 Russia.
Genetika. 2001 Aug;37(8):1104-11.
Based on originally designed technique of myoblast cultivation and in accordance with the approved by the Russian Ministry of Health "one muscle treatment" protocol of myoblast transplantation to the Duchenne muscular dystrophy patients, the first in Russia clinical trial of this gene correction method was carried out. Immonologically related myoblast cultures (30 to 90 million cells per patient) were injected after all preliminary procedures into tibialis anterior muscles of four boys selected from a group of volunteer recipients (Duchenne muscular dystrophy patients) based on the analysis of a number of surface antigens in donor-recipient pairs. The condition of the patients remained satisfactory during the whole period of post-transplantation follow-up (from 6 months to 1.5 years). Six months after myoblast transplantation the presence of donor DNA or dystrophin synthesis was demonstrated in muscle biopsies of three out of four patients. This result confirms efficacy and safety of the procedure used.
基于最初设计的成肌细胞培养技术，并按照俄罗斯卫生部批准的针对杜氏肌营养不良患者的“单肌肉治疗”成肌细胞移植方案，在俄罗斯首次开展了这种基因矫正方法的临床试验。在完成所有初步程序后，根据供体-受体对中多种表面抗原的分析，从一组志愿者受者（杜氏肌营养不良患者）中挑选出4名男孩，将免疫相关的成肌细胞培养物（每名患者3000万至9000万个细胞）注射到他们的胫前肌中。在移植后的整个随访期（6个月至1.5年）内，患者的状况一直令人满意。成肌细胞移植6个月后，在4名患者中的3名患者的肌肉活检中证实存在供体DNA或抗肌萎缩蛋白合成。这一结果证实了所用程序的有效性和安全性。
