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GENE THERAPY By Sam Lai and Meng Lee Gene Therapy ? Introduction : What is it? ? Gene Therapy : A Closer Look ? How much did you learn?! ? Case Study : SCID ? Model for Gene Therapy Medical Framework ? Discussion / Qamp。A What is it? ? Gene therapy, which was first conceived in the mid1970s, aims to insert working copies of genes into cells containing dysfunctional ones ? treat, cure, or ultimately prevent disease by changing the expression of a person39。s genes ? current gene therapy is primarily experimental, with most human clinical trials only in the research stages ? Most often in context of treating lethal and disabling diseases, potential also for disease prevention Human Genome Project ? A genome is all the DNA in an anism, including its genes. Genes carry information for making all the proteins required by all anisms ? Variations in structure of person’s genes collectively helps define us as individuals ? Rationale for GT is based on knowledge of the human geic code ? Began formally in 1990, set as a 13 year project. HGP GOALS ? Identify aproximately 30,000 genes ? Determine 3 billion chemical base pairs that make up human DNA ? Store information in databases ? Improve tools for data analysis ? Address ethical, legal and social issues that might arise from this HGP cont… ? Achieve these goals, researchers look at geic makeup of other anisms ? This project is important because the government39。s dedication to the transfer of technology to the private sector Which Cells Targeted? ? somatic gene therapy the recipient39。s genome is changed, but the change is not passed along to the next generation ? 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 a lot of discussion is being conducted about its value and desirability. Somatic Stem Cells ? Somatic stem cells are remarkable cells in the body ? These different stem cell types are targets for gene therapy ? Geically treated stem cells, when reintroduced into the patient39。s body are expected to naturally travel through the b