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Updated: Apr 4, 2026
Implementing Patch Clamp and Live Fluorescence Microscopy to Monitor Functional Properties of Freshly Isolated PKD Epithelium
Published on: September 1, 2015
Therapeutic targets for polycystic kidney disease
1a University College of London, Royal Free Hospital, UCL Centre for Nephrology , Rowland Hill Street, London, NW3 2PF, UK.
Introduction:
Polycystic kidney disease (PKD) is a common genetic disease in which renal enlargement and loss of function is caused by progressive expansion of tubular cysts. To reverse the detrimental effects of PKD gene mutation(s) and to slow cystic expansion, new drug therapies are required.
Areas Covered:
The underlying cell biology leading to identification of molecular targets for PKD is reviewed. Specific focus is on studies published at the early pre-clinical level. These include genetic and epigenetic modulators, and drugs to slow cystic expansion and disease progression. Discussion of specific drugs and clinical trials is not within the scope of this article. Literature research methods included EndNote and PubMed online searches using keyword combinations: polycystic kidneys disease, pre-clinical, molecular targets, signal transduction, genetic modulators, epigenetic, therapeutic, receptors, kinases. Where possible, the most recent citations concerning a given target are referenced.
Expert Opinion:
It is suggested that the most promising targets for future therapeutic development are those that target upstream signaling events at cell membranes, such as the vasopressin-2 receptor (AVPR2), EGFR/ErbB2, and the β-1-integrin receptor, as well as the intracellular integrator kinase, c-Src.
Insights
New drug therapies are needed for polycystic kidney disease (PKD). Promising pre-clinical targets include cell membrane receptors and intracellular kinases to slow disease progression.
Area of Science:
- Nephrology
- Genetics
- Pharmacology
Background:
- Polycystic kidney disease (PKD) is a common genetic disorder characterized by progressive renal enlargement and cyst formation.
- This leads to significant loss of kidney function, necessitating novel therapeutic strategies.
Purpose of the Study:
- To review the underlying cell biology of PKD for identifying molecular targets.
- To focus on pre-clinical studies of genetic, epigenetic, and signaling pathway modulators for therapeutic development.
Main Methods:
- Literature search using EndNote and PubMed with keywords related to PKD, pre-clinical research, molecular targets, and therapeutic strategies.
- Review of studies focusing on genetic and epigenetic modulators and drugs targeting signal transduction pathways.
Main Results:
- Identified several key molecular targets involved in PKD pathogenesis.
- Highlighted the potential of targeting upstream signaling events at cell membranes and intracellular kinases.
Conclusions:
- The vasopressin-2 receptor (AVPR2), EGFR/ErbB2, and β-1-integrin receptor are promising therapeutic targets.
- The intracellular kinase c-Src also represents a significant target for future PKD drug development.
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Major types that are helpful drug targets include: