基因治疗-复旦《医用分子遗传学》课件.pdf

Gene Therapy 基因治疗 复旦大学出生缺陷研究中心 复旦大学分子医学教育部重点实验室 马端 Gene Therapy What is Gene Therapy? Gene therapy is an experimental treatment that involves introducing genetic material into a person’s cells to fight or prevent disease. History of Gene Therapy  First unapproved gene therapy – 1980, Dr. Martin Cline, bone marrow cells of two patients with beta-thalassemia. UCLA First human somatic gene therapy – 1989, four year old Ashanti Desilva with SCID (Adenosine deaminase deficiency) First complete gene therapy cures, 2000, France, 10 children with X-SCID, Bubble Boy. In 2001, researchers at St. Barnabas Hospital in New Jersey announced that they had used ooplasmic transfer to enable several women with impaired fertility to bear children. Predicament  1999, Jesse Gelsinger died after gene therapy for an inherited liver disease called ornithine transcarbamylase deficiency (鸟氨酸氨甲酰转移酶缺乏症).  3 of the 11 infants that were successfully treated in Paris developed leukemia between 3 and 6 years after the treatment.  36-year-old Illinois woman undergoing treatment for rheumatoid arthritis died in 2007. Clinical Trial World Wide Gene Therapy In China  1987: 薛京伦,卢大儒。血友病B，1991年治疗4例，17年缓解，逆转录病毒。 2003年获第二个临床批件。重组AAV-2人凝血因子IX注射液。  2004: 重组人p53腺病毒注射液（今又生），头颈鳞癌 。 世界上第一个基因治疗上市产品，深圳赛百诺基因技术有限公司。  2005：第一个具有溶瘤作用的重组人5型腺病毒（H101）与化疗结合治疗难 治性晚期鼻咽癌（安柯瑞），上海三维生物技术有限公司。 Basic Process of Gene Therapy Replacing a mutated gene that causes disease with a healthy copy of the gene Inactivating, or “knocking out,” a mutated gene that is functioning improperly Introducing a new gene into the body to help fight a disease Key Points of Gene Therapy Normal Gene or RNA