How to identify a target: An overview

Welcome to the week 3: how to identify a target. My name is Ming-Heng Wu, an assistant professor in graduate institute of translational medicine of Taipei Medical University. In the week 2, you have learned most of disease are caused by the gene mutations or changed gene expression. If you want to develop a drug for a disease, you need to identify which gene is the key regulator for the disease progression. These genes, we call them target. Which means they will be the therapeutic targets for the disease and for future drug development. As you all know that the central dogma of gene expression is from DNA to RNA and from RNA to Proteins.

Any alterations in each level will change the final protein functions and expression. In addition, there are at least 30,000 genes encoded in our genome. Therefore, this week, we will start from several high throughput screening

methods from three aspects: genomics, transcriptomics, and proteomics which analyzes the DNA, RNA, and protein samples from healthy individuals and patients. These methods allows us simultaneously analyze the change of large number of genes in one experiment. Finally, after we identify the disease associated genes, the next step is to identify which gene is the real target of the disease. We call this process target validation. This is because not all of the disease associated genes affect disease progression. Many of the gene changes are acquired from the disease rather than cause the disease. So in the last part of this course, we will focus on how to do target validation and also we will provide you an example.

Hopefully we can stimulate new ideas and please feel free to discuss with our teams. Enjoy this week.