Can genetic diseases be cured?
The answers turns out to be: it depends.
Today we explored gene therapy, the process of adding a functioning gene to a patient's cells and/or blocking a harmful gene. We determined whether different diseases would be good candidates for gene therapy by answering four important questions:
Do we know which tissues and proteins are affected?
Are only 1-2 genes working incorrectly?
Would a correct gene copy fix the disease, or does a faulty gene also need to be blocked? and
Can we deliver the gene to affected cells?
One by one, we examined whether gene therapy can cure cystic fibrosis, sickle cell disease, Wilson's disease, and Down syndrome. By applying what we knew about how genes affect proteins, we were able to distinguish between strong and weak candidates for gene therapy! We also considered different methods of delivering genes to cells, such as in vivo vs. ex vivo delivery, and gene delivery using viral vectors.