摘要
为了探讨干细胞移植 (StemCellTransplantation)对神经系统中预后不良的单基因遗传性疾病之一DMD(假肥大型肌营养不良症 )的临床可行性 ,采用PCR反向序列特异性寡核苷酸杂交技术 ( polymerasechainreaction reversesequencespecificoligonucleotide ,PCR RSSO)对 3 0例DMD患者进行HLA ABDR基因分型 ,并与广东省脐血干细胞库 668份和中国造血干细胞捐献者库 3 4 910份分别进行HLA ABDR基因频率比较和无关供者干细胞的配合性分析。结果表明 :DMD疾病组的HLA基因呈正态遗传 ,DMD患者HLA基因表达与广东健康人群无统计学上显著差异 ;DMD患者中 1/ 4寻找到合适用于移植的干细胞 ,其中在广东脐血库寻找到≥ 5个HLA ABDR基因配合的几率为 5 0 % ,在中国造血干细胞库寻找到 6个HLA ABDR基因配合的几率为 2 6%。结论 :对DMD患者进行非亲缘异基因脐带血 /外周血移植治疗具有HLA配合供者高选择几率的可行性基础 ;干细胞移植不仅将使致死性神经肌肉疾病患者获得临床症状改善 ,并且为难治性神经系统疾病受益于干细胞移植治疗打造技术平台。
The aim of this study was to investigate the clinical feasibility of adult stem cell transplantation for lethal mono gene inherited disease, Duchenne muscular dystrophy (DMD). A total of 30 blood samples from DMD patients were genotyped with HLA A, B and DR alleles by means of polymerase chain reaction reverse sequence specific oligonucleotide (PCR RSSO). The HLA gene types in 30 DMD patients were compared with those of 668 unrelated donors from Umbilical Cord Blood Center of Guangdong Province and 34 910 unrelated donors from Chinese Bone Marrow Bank. The results showed that HLA gene of the DMD group was inherited in normal distribution. There was no striking difference of HLA A, B and DR alleles expression between the DMD patients group and control healthy group. 25% of the DMD patients got suitable donors for stem cell transplantation, in which 15 patients found donors with ≥5/6 HLA match at the Umbilical Cord Blood Center of Guangdong Province, i.e. occupying 50% of the total. Eight patients got 6/6 HLA matching donors at the Chinese Bone Marrow Bank, i.e. occupying 26% of the total. It is concluded that stem cells transplantation therapy for DMD patients is feasible, which will benefit these patients suffered from the lethal neuromuscular disease, and create a new way to treat this tough nervous system disease.
出处
《中国实验血液学杂志》
CAS
CSCD
2004年第6期845-848,共4页
Journal of Experimental Hematology
