医学遗传学 19遗传疾病的治疗eng v.ppt

19 基因治疗 Gene Therapy;1. What is gene therapy? ;;;; Researchers may use one of several approaches for correcting faulty genes: A normal gene may be inserted into a nonspecific location within the genome to replace a nonfunctional gene. This approach is most common. An abnormal gene could be swapped for a normal gene through homologous recombination. The abnormal gene could be repaired through selective reverse mutation, which returns the gene to its normal function. The regulation (the degree to which a gene is turned on or off) of a particular gene could be altered. ;2. How does gene therapy work? ;;;;;;To reverse disease caused by genetic damage, researchers isolate normal DNA and package it into a vector, a molecular delivery truck usually made from a disabled virus. Doctors then infect a target cell —usually from a tissue affected by the illness, such as liver or lung cells—with the vector. The vector unloads its DNA cargo, which then begins producing the missing protein and restores the cell to normal.;;(2)Adenoviruses A class of viruses with double-stranded DNA genomes that cause respiratory, intestinal, and eye infections in humans. The virus that causes the common cold is an adenovirus. (3)Adeno-associated viruses A class of small, single-stranded DNA viruses that can insert their genetic material at a specific site on chromosome 19. (4)Herpes simplex viruses A class of double-stranded DNA viruses that infect a particular cell type, neurons. Herpes simplex virus type 1 is a common human pathogen that causes cold sores. ;降穗验警薛量贴侥忙还茁开赛宅壹摧远拌抱湃构睁疏物藤蓄孔宵沿夫讯挥医学遗传学 19遗传疾病的治疗eng v医学遗传学 19遗传疾病的治疗eng v;;;;;;;;;3. The current status of gene therapy research;;;;;;;4. Factors kept gene therapy effective;;;;;;;;;;5. Some recent developments ;;;;;;6. Some of the ethical considerations ;