Emery Alan E H
Department of Neurology, Royal Devon and Exeter Hospital, EX2 5DW, Exeter, UK.
Neuromuscul Disord. 2002 May;12(4):343-9. doi: 10.1016/s0960-8966(01)00303-0.
Since the identification of the gene for Duchenne muscular dystrophy and its protein product some 15 years ago, the basic defects in all the commoner forms of dystrophy have now been identified. It is thus possible, on the basis of this information, to make a precise diagnosis in an affected individual and to offer accurate genetic counselling and prenatal diagnosis. Now newer technologies are being applied to the investigation of these disorders. These include studies of single nucleotide polymorphisms, microarray analysis and expression profiling, the yeast two-hybrid assay, and proteomics. A great deal of new information is emerging in this way which will hopefully help us to understand the causes of inter-familial and intra-familial variation and particularly pathogenesis, a detailed understanding of which could be the first step in finding effective treatments.
大约15年前,自杜氏肌营养不良症的基因及其蛋白质产物被鉴定以来,现在已经明确了所有较常见形式的肌营养不良症的基本缺陷。因此,基于这些信息,有可能对受影响个体进行精确诊断,并提供准确的遗传咨询和产前诊断。现在,更新的技术正在应用于这些疾病的研究。这些技术包括单核苷酸多态性研究、微阵列分析和表达谱分析、酵母双杂交试验以及蛋白质组学。通过这种方式正在涌现大量新信息,有望帮助我们理解家族间和家族内变异的原因,尤其是发病机制,对其详细了解可能是找到有效治疗方法的第一步。