Mesoscale Nanotechnology: A Novel Therapeutic Strategy in Polycystic Kidney Disease
Abstract
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, 2022
- Accession Number
- AD1169500
Entities
People
- Edgar A. Jaimes
Organizations
- Memorial Sloan Kettering Cancer Center