Gene Therapy to cure Autosomal Dominant Polycystic Kidney Disease (Cure-ADPKD)

• Applicants: Professor Dr. Rafael Kramann; Professor Dr.-Ing. Jørgen Magnus
• Subject Area: Nephrology
• Term: since 2025
• Project identifier: Deutsche Forschungsgemeinschaft (DFG) - Project number 561848128

Disease (Cure-ADPKD) The prevalence of chronic kidney disease (CKD) has reached >10% worldwide, and CKD globally accounts for more years lived with disability (YLD) than all neoplasms combined. Currently, available “best” CKD therapies are limited, non-specific, and of poor efficacy. Therefore, there is an urgent medical, social, and economic need to develop novel therapeutics for chronic kidney disease. Autosomal polycystic kidney disease (ADPKD) is the second most common autosomal dominant disease in the world and the most common hereditary kidney disease. ADPKD accounts for 10% of all dialysis patients in Germany and the treatment options for this devastating disease are particularly limited. The recently approved therapy with tolvaptan leads to a reduction in cyst growth and, in the best case, to an extension of the dialysis-free time by a few years but is associated with enormous side effects. In the planned study, we will address this issue and will establish a platform to develop gene therapy strategies to treat ADPKD. RWTH Aachen university is a technical university including a medical faculty. This combination of engineering, natural and medical sciences is unique in Germany. The planned project CureADPKD takes advantage of this and will join the expertise of two scientists from different faculties (medicine and bioprocess engineering) to develop a new therapy for treatment of ADPKD.

• DFG Programme: Research Grants