The Genetics Mutations
There are three genetic mutations in the PKD-1, PKD-2, and PKD3 gene with similar phenotypical presentations. Given that PKD is one of the most common hereditary diseases in the United States, is there any plans out there for testing gene therapy and CRISPR methods as a treatment for this common genetic disease?
Gene editing may be most practical in hematology, whereby stem cells can be isolated, expanded, and edited and then reintroduced into the patient. With differentiated cells, getting the editing vectors into all cells is a major hurdle.