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Updated: Jan 2, 2026

Use of Ultra-high Field MRI in Small Rodent Models of Polycystic Kidney Disease for In Vivo Phenotyping and Drug Monitoring
Published on: June 23, 2015
A no-nonsense approach to hereditary kidney disease
Fatima Tokhmafshan1, Kyle Dickinson1,2, Murielle M Akpa1
1Research Institute of the McGill University Health Center, 1001 Décarie Boulevard, EM1.2232, Montreal, QC, H4A 3J1, Canada.
Abstract:
The advent of a new class of aminoglycosides with increased translational readthrough of nonsense mutations and reduced toxicity offers a new therapeutic strategy for a subset of patients with hereditary kidney disease. The renal uptake and retention of aminoglycosides at a high intracellular concentration makes the kidney an ideal target for this approach. In this review, we explore the potential of aminoglycoside readthrough therapy in a number of hereditary kidney diseases and discuss the therapeutic window of opportunity for subclasses of each disease, when caused by nonsense mutations.
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