Mesoscale Nanotechnology: A Novel Therapeutic Strategy in Polycystic Kidney Disease
Abstract
The central hypothesis of this proposal is that the targeted tubular delivery of novel therapeutic agents is an effective strategy for the treatment of polycystic kidney disease. Aim 1. Evaluate therapeutic efficacy of nanoparticle-targeted mTOR inhibition in a rat model of PKD Mesoscale nanoparticles will be synthesized to encapsulate an ASO against mTOR. We will use the PCK-Pkhd1pck rat model of PKD that closely mimics ADPKD in humans. Efficacy will be evaluated by longitudinal serial live imaging via MRI, as well as post-mortem phenotypic surrogate markers of renal cystogenesis. Aim 2. Evaluate therapeutic efficacy of nanoparticle-targeted miR-17 inhibition in a rat model of PKD Mesoscale nanoparticles will be synthesized to encapsulate an oligonucleotide against miR-17. Just like in Aim 1 we will use the Pkhd1pck/pck rat model of PKD that closely mimics ADPKD in humans. Efficacy will be evaluated by longitudinal serial live imaging via MRI, as well as post-mortem phenotypic surrogate markers of renal cystogenesis.
Document Details
- Document Type
- Technical Report
- Publication Date
- May 01, 2023
- Accession Number
- AD1214486
Entities
People
- Edgar A. Jaimes
Organizations
- Memorial Sloan Kettering Cancer Center