Therapeutic Genome Editing (PHA 4026)
(05/03/2021-07/30/2021)
Course Memo
New therapies that employ genome editing tools are being developed for a broad range of diseases that include sickle-cell disease, beta-thalassemia, Duchenne muscular dystrophy and others. The effectiveness of these genome-based therapies is evident from the case of a patient with sickle-cell disease who underwent treatment that employed genome editing, resulting in remarkable remission from the disease. Encouraged by this success, genome editing technologies are being developed for a large number of diseases, and some of which are already in Phase I/II trials. Some of the diseases are viral infections (hepatitis/HPV), cardiovascular disease, cystic fibrosis, metabolic disorders, defects of the immune system, hemophilia, muscular dystrophy, and development of T cell-based anticancer immunotherapies, all of which are familiar to a PharmD student.