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Treatment prospects for autosomal-dominant polycystic kidney disease
Q Qian1, P C Harris, V E Torres
1Mayo Clinic/Mayo Foundation, Rochester, Minnesota 55905, USA.
Abstract:
An increased understanding of the molecular genetic and cellular pathophysiologic mechanisms responsible for the development of autosomal-dominant polycystic kidney disease (ADPKD), made possible by the advances in molecular biology and genetics of the last three decades, has laid the foundation for the development of effective therapies. As the concept that a polycystic kidney is a neoplasm in disguise is becoming increasingly accepted, the development of therapies for ADPKD may benefit greatly from the expanding body of information on cancer chemoprevention and chemosuppression. This review summarizes the observations that already have been made and discusses therapies for PKD that deserve investigation.
Insights
Advances in understanding autosomal-dominant polycystic kidney disease (ADPKD) mechanisms pave the way for new therapies. Research into cancer chemoprevention may offer novel treatment strategies for ADPKD.
Area of Science:
- Molecular biology and genetics
- Nephrology
- Oncology
Background:
- Recent advances in molecular biology and genetics have significantly improved understanding of autosomal-dominant polycystic kidney disease (ADPKD) pathogenesis.
- The concept of polycystic kidneys as neoplasms is gaining traction, suggesting potential therapeutic parallels with cancer research.
Purpose of the Study:
- To review current understanding of ADPKD molecular and cellular mechanisms.
- To explore the potential of cancer chemoprevention and chemosuppression strategies for ADPKD therapy development.
Main Methods:
- Literature review of recent findings in ADPKD molecular genetics and pathophysiology.
- Analysis of established cancer chemoprevention and chemosuppression principles for potential application to ADPKD.
Main Results:
- Significant progress in elucidating the genetic and cellular underpinnings of ADPKD.
- Identification of potential therapeutic targets and strategies informed by cancer research.
Conclusions:
- The foundational knowledge of ADPKD mechanisms supports the development of targeted therapies.
- Investigating cancer chemoprevention and chemosuppression approaches holds promise for novel ADPKD treatments.