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基因治疗方法是通过基因转导技术赋于靶细胞一种新的功能或改变靶细胞某些基因的表达状况。目前基因治疗的疾病主要包括遗传性疾病、免疫缺陷病、肿瘤等。1991年12月在复旦大学遗传学研究所进行了国际上首例血友病的基因治疗。两位由于凝血因子Ⅸ缺乏而患有血友病B的患者被重新植入了大量转移有正常Ⅸ因子基因的自身皮肤成纤维细胞,结果病人体内Ⅸ因子含量都成倍上升,取得较好疗效。1990年9月,美国国立卫生研究院(NIH)用基因疗法治疗了一位由于体内腺苷脱氨酶(ADA)缺乏而患者严重联合性免疫缺陷的病人,他们将转移有正常ADA基因的患者自身淋巴细胞体外培养后重新大量输回病人体内,实验结果令人鼓舞。目前基因治疗已应用于很多种疾病。 肿瘤的基因治疗始于九十年代。1990年美国NIH首次对一位晚期恶性黑色素瘤病人进行了基因转导的肿瘤浸润淋巴细胞(TIL)
Gene therapy methods impart a new function to target cells or change the expression of certain genes in target cells through gene transduction technology. Current gene therapy diseases include hereditary diseases, immunodeficiency diseases, and tumors. In December 1991, the first international gene therapy for hemophilia was performed at the Institute of Genetics at Fudan University. Two patients with hemophilia B due to the deficiency of coagulation factor IX were re-implanted with a large number of self-transplanted skin fibroblasts with a normal factor VIII gene. As a result, the factor VIII levels in the patients both increased exponentially and achieved better results. . In September 1990, the National Institutes of Health (NIH) used gene therapy to treat a patient with severe combined immunodeficiency due to lack of adenosine deaminase (ADA) in the body. They will transfer patients with normal ADA genes. After the in vitro culture of autologous lymphocytes was reintroduced back into the patient, the experimental results were encouraging. At present, gene therapy has been applied to many diseases. Gene therapy for tumors began in the 1990s. In 1990, the United States NIH conducted a gene-transformed tumor-infiltrating lymphocyte (TIL) for the first time in a patient with advanced malignant melanoma