Ozawa K
Division of Hematology, Department of Medicine, Jichi Medical School.
Nihon Rinsho. 2001 Jan;59(1):59-64.
With advances in DNA chip(DNA microarray) technology, it has become possible to obtain genome-wide gene-expression profiling. This novel technology is now applied to the study of molecular pathogenesis, differential diagnosis, and prediction of prognosis in the field of hematological malignancies. Importantly, the list of informative genes includes new markers of diseases, which will be utilized for further investigation. As for clinical gene therapy, it has been criticized for promising too much and providing too little during the last 10 years. In 1999, however, the success of hematopoietic stem cell gene therapy was reported in the patients with X-SCID(X-linked severe combined immunodeficiency). The gene therapy of hemophilia B using AAV(adeno-associated virus) vector has also successfully started. Genetic manipulation will be applied to the treatment of various diseases with the development of novel technologies.
随着DNA芯片(DNA微阵列)技术的进步,获得全基因组基因表达谱已成为可能。这项新技术目前已应用于血液系统恶性肿瘤领域的分子发病机制研究、鉴别诊断及预后预测。重要的是,信息基因列表中包括疾病的新标志物,这些标志物将用于进一步研究。至于临床基因治疗,在过去十年中,它因承诺过多而实际成效过少受到批评。然而,1999年,有报道称X连锁重症联合免疫缺陷症(X-SCID)患者的造血干细胞基因治疗取得成功。使用腺相关病毒(AAV)载体的B型血友病基因治疗也已成功开展。随着新技术的发展,基因操作将应用于各种疾病的治疗。