1、GENE THERAPY By Sam Lai and Meng LeeGene Therapyn Introduction : What is it?n Gene Therapy : A Closer Lookn How much did you learn?!n Case Study : SCIDn Model for Gene Therapy Medical Frameworkn Discussion / Q&AWhat is it?n Gene therapy, which was first conceived in the mid-1970s, aims to insert wor
2、king copies of genes into cells containing dysfunctional ones n treat, cure, or ultimately prevent disease by changing the expression of a persons genes n current gene therapy is primarily experimental, with most human clinical trials only in the research stages n Most often in context of treating l
3、ethal and disabling diseases, potential also for disease preventionHuman Genome Projectn A genome is all the DNA in an organism, including its genes. Genes carry information for making all the proteins required by all organisms n Variations in structure of persons genes collectively helps define us
4、as individualsn Rationale for GT is based on knowledge of the human genetic code n Began formally in 1990, set as a 13 year project. HGP GOALS n Identify aproximately 30,000 genes n Determine 3 billion chemical base pairs that make up human DNAn Store information in databasesn Improve tools for data
5、 analysisn Address ethical, legal and social issues that might arise from thisHGP contn Achieve these goals, researchers look at genetic makeup of other organismsn This project is important because the governments dedication to the transfer of technology to the private sector Which Cells Targeted? n
6、 somatic gene therapy the recipients genome is changed, but the change is not passed along to the next generation n germline gene therapy, the sex cells are changed with the goal of passing these changes to their offspring. But, this is not being actively investigated, at least not humans, although
7、a lot of discussion is being conducted about its value and desirability. Somatic Stem Cells n Somatic stem cells are remarkable cells in the bodyn These different stem cell types are targets for gene therapyn Genetically treated stem cells, when reintroduced into the patients body are expected to naturally travel through the blood stream to the bone marrow.
