This interactive module uses the central dogma as a model for exploring how modern molecular biology technologies can be used to treat different genetic conditions.

The Click & Learn describes different steps in the pathways from gene to RNA to protein. It also provides information about a treatment strategy that targets each step, and an example of a genetic disease for which that strategy has been shown to work, either in experimental models or human clinical trials. The examples include treatments for cystic fibrosis, sickle cell disease, and Huntington’s disease, and are supplemented by video clips from the documentary The Gene Doctors, an HHMI Tangled Bank Studios Production.

The accompanying worksheet guides students’ exploration.