GENETHERAPY-Deepwater：基因疗法-深水.pptVIP

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 / QA What is it? Gene therapy, which was first conceived in the mid-1970s, aims to insert working copies of genes into cells containing dysfunctional ones treat, cure, or ultimately prevent disease by changing the expression of a persons 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 organism, including its genes. Genes carry information for making all the proteins required by all organisms Variations in structure of person’s genes collectively helps define us as individuals Rationale for GT is based on knowledge of the human genetic 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 genetic makeup of other organisms This project is important because the governments dedication to the transfer of technology to the private sector Which Cells Targeted? somatic gene therapy the recipients 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 Gene