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08:46
Implementing Patch Clamp and Live Fluorescence Microscopy to Monitor Functional Properties of Freshly Isolated PKD Epithelium
Published on: September 1, 2015
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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.
Expert Opinion on Therapeutic Targets
|September 3, 2015
Summary
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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