Question

hhmi Bieinteractive Activity Central Dogma Card Sorting Activity Student Handout 4. Another disease caused by a mutation in a single gene is Huntington's disease (HD), an autosomal dominant condition. It is caused by mutations in a gene required for normal nerve cell function. The mutations cause abnormal proteins to be produced which "stick together and accumulate in nerve cells, eventually interfering with normal cell operations. Suggest two ways you could treat the disease by targeting the translation step for the HD protein and justify why each approach might be effective. 5. (Optional) For any genetic disease, several approaches for treating it at different steps of gene expression could work. Complete the Click & Learn "Central Dogma and Genetic Medicine" paying particular attention to the genetic medicines that have been developed or are in development for the diseases above. Were the approaches you identified in this activity like the ones in the interactive? If so, how were they similar? If not, how did they differ? 6. Consider hemophilia again. Identify two ways the researcher could design an intervention to treat hemophilia, provide a brief explanation of each, and justify why each approach might be effective. Genetics