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Published on: September 1, 2015
Gene therapy for kidney disease: targeting cystinuria
Jennifer L Peek1,2, Matthew H Wilson2,3,4,5
1Medical Scientist Training Program, Vanderbilt University School of Medicine.
Purpose Of Review:
The aim of this study was to summarize recent findings in kidney gene therapy while proposing cystinuria as a model kidney disease target for genome engineering therapeutics.
Recent Findings:
Despite the advances of gene therapy for treating diseases of other organs, the kidney lags behind. Kidney-targeted gene delivery remains an obstacle to gene therapy of kidney disease. Nanoparticle and adeno-associated viral vector technologies offer emerging hope for kidney gene therapy. Cystinuria represents a model potential target for kidney gene therapy due to its known genetic and molecular basis, targetability, and capacity for phenotypic rescue.
Summary:
Although gene therapy for kidney disease remains a major challenge, new and evolving technologies may actualize treatment for cystinuria and other kidney diseases.
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