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?
The short answer is no. It seems unlikely even in the long run because trying to repair or alter the mutant gene in an adult kidney is impractical. Even if you could identify the mutation in every patient, trying to correct this in every renal epithelial cell is just not feasible. 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.
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