Health Restored
Programs For Polycystic Kidney Disease
Polycystic Kidney Disease (PKD) is a genetic disorder that causes cysts to slowly and progressively grow in the kidneys. These cysts can be painful and damage the kidney, often requiring dialysis or transplant.
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RESTARI is developing a well-tolerated small molecule taken as a once-a-day pill, that shrinks PKD cysts and reduces large cyst formation.
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RESTARI is researching PKD gene therapies delivered directly into the kidney using our AKI device. This gene therapy is intended to repair the genetic disorder that causes PKD.
Image Source: National Institute of Diabetes and Digestive and Kidney Diseases
Frequency of Cysts Compared to Cyst Volume After Treatment
Higher doses of RESTARI's compound reduced the occurrence of large PKD cysts in vitro. (RESTARI Data on File)
A demonstration of RESTARI's capability to deliver gene therapies into renal cells (3).
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These data provide signs of intact renal structural and function capacities post hydrodynamic transgene delivery. The data are taken from a live rat 3 days after it was treated with pEGFP and pEGFP-Actin naked plasmid vectors. Images (A-C) outline pEGFP and (D-F) outline pEGFP-Actin transgene expression in proximal tubule (PT) epithelial cells. Solutions containing 3kDa Cascade blue and 150 kDa TRITC dextrans were infused into the jugular veins of live rats. Robust and widespread uptake of the low molecular weight dextran dextran solutions was observed after dye infusion, presented in images (B) and (E). The Cascade blue dextran was rapidly filtered by glomeruli, and was then endocytosed by into proximal tubule epithelial cells. Additionally, the large molecular weight dextran molecules were restricted to the vasculature as shown in images (C) and (F). Images (C and F) are the mergers of the green, blue and red pseudo-colors originating from the EGFP transgenes, and low and large molecular weight dextrans respectively.
Valen Keefer, PKD patient. According to the National Kidney Foundation
“Kidney disease and post transplant patients can live a joyful, productive and fulfilling life. Healing is not linear and at times can be a long process. Be kind to yourself through it all. Mental wellness, self-love, and acceptance is so important.”
Bibliography
​(3)A Method to Facilitate and Monitor Expression of Exogenous Genes in the Rat Kidney Using Plasmid and Viral Vectors. Corridon, Bacallao, Basile, Atkinson et al. Am J Physiol Renal Physiol 304: F1217-F1229, 2013.