浅谈基因治疗及其发展历史 黄慧敏 515150910054
浅谈基因治疗及其发展历史 黄慧敏 515150910054
基因治疗(gene therapy) ■将外源正常基因导入靶细胞，以纠正或补偿因基因缺陷和异常 引起的疾病，以达到治疗目的。 ·原位修复有缺陷的基因 。用正常基因替代缺陷基因
基因治疗（gene therapy） 将外源正常基因导入靶细胞，以纠正或补偿因基因缺陷和异常 引起的疾病，以达到治疗目的。 原位修复有缺陷的基因 用正常基因替代缺陷基因
应用 一基因治疗主要是治疗那些对人类健康威胁严重的疾病，包括： 一遗传病（如血友病、囊性纤维病、家庭性高胆固醇血症等） ●恶性肿瘤 ●心血管疾病 。感染性疾病（如艾滋病、 类风湿等) 业M
应用 基因治疗主要是治疗那些对人类健康威胁严重的疾病，包括： 遗传病（如血友病、囊性纤维病、家庭性高胆固醇血症等） 恶性肿瘤 心血管疾病 感染性疾病（如艾滋病、类风湿等）
发展历史 一1963年，美国分子生物学家、诺贝尔生理学或医学奖获得者乔 舒亚莱德伯格(Joshua Lederberg)提出了基因交换和基因 优化的理念。 ●1970年，美国医生斯坦菲尔德·罗杰斯(Stanfield Rogers)试 图通过注射含有精氨酸酶的乳头瘤病毒来治疗一对姐妹的精氨 酸血症，试验失败了
发展历史 1963年，美国分子生物学家、诺贝尔生理学或医学奖获得者乔 舒亚·莱德伯格（Joshua Lederberg）提出了基因交换和基因 优化的理念。 1970年，美国医生斯坦菲尔德·罗杰斯（Stanfield Rogers）试 图通过注射含有精氨酸酶的乳头瘤病毒来治疗一对姐妹的精氨 酸血症，试验失败了
发展历史 ●1972年，美国著名生物学家西奥多·弗里德曼(Theodore Friedmann)等人在《科学》(Science)杂志上发表评论 《基因治疗能否用于人类遗传病？》 proaucea no narmrut enects on re. Gene Therapy for Human Genetic Disease? peated blind passages in tissue culture. The virus was tested immunologically In contrast to Friedmann and Rob. patient's circulation.The quantity of for possible contamination by other lin.who state."Storage diseases enzyme was far too small to expect a viruses,and none was found.Ulti- associated with lysosomal enzyme de- notable therapeutic benefit,but could mately it was filtered through Millipore ficiencies do not appear to respond to not be increased because of the dele- filters. enzyme therapy"(1).we believe that terious effect of administering large The virus has been studied for 40 there is considerable hope for treat- amounts of plasma to Fabry patients. years without having produced any ment of some lysosomal enzyme defi- Clearly,the experiment gives no in- known harmful effects on any investi- ciencies by this means.This is so formation on the therapeutic potential of purified enzyme supplied in appro. gator.including one who inoculated because proteins can be transported himself in 1933.Massive doses have into cells through the process of pino. priate dosage. been given to many kinds of animals cytosis.followed by a merger of the ELIZARETH F.NEUFELD without discernible effect except for. pinocytic vesicles with lysosomes and a National Institute of Arthritis and as pointed out,the decrease in blood mixing of their contents.Therapeutic Metabolie Diseases. arginine concentration.Inoculated into replacement of deficient emzymes with National Institutes of Health. the skin of wild or domestic rabbits. plasma.leukocytes,or through trans- Bethesda,Maryland 20014 it produces warts.The wild-type virus planted kidneys (rather than with puri. CHARLES C.SWEELEY i nat nronsaahle in the domestic rah-
发展历史 1972年，美国著名生物学家西奥多·弗里德曼（Theodore Friedmann）等人在《科学》（Science）杂志上发表评论 《基因治疗能否用于人类遗传病？》