ILOs

The student should know about, e.g.

• some of the approaches that will be introduced or expanded in the near future
• know about the relevant principles of pharmacogenetics
• gene therapy,
• exon-skipping
• induced read-through of stop codons
• gene editing & CRISPR-based technologies
• examples of current applications of precision medicine for inherited clinical genetic conditions and the likely future expansion of these types of therapies

• What does the future of treatments for inherited disorders hold?
• What’s one of the problems with drugs in general right now…?
• What is precision medicine?
• What are the strategies in gene therapy?
• Outline some new/future therapies for DMD & other disorders…
• Summary of some recent gene therapy approaches