Gene transfer to the kidney remains understudied. This is unfortunate as chronic kidney disease plagues up to 14% of the U.S. population. Dialysis remains the mainline treatment for end stage renal disease (ESRD) as kidney transplants are very limited given organ availability. The mortality of patients on dialysis (5 year survival ~20% for patients with ESRD and diabetes) is equivalent if not worse than many cancers which receive quite a bit of support for cell and gene therapy approaches. Gene transfer approaches that could cure or limit progression of kidney disease would have high impact on eliminating the widespread mortality and morbidity associated with kidney disease.
We use a combination of approaches to attack kidney disease including:
- nonviral (transposons)
- viral (adeno-associated viruses)
- genome editing (CRISPR/Cas9)
- new technologies
- animal models of inherited kidney diseases