Minimally-Invasive Therapy for Parkinson’s Disease Achieved by the Focused Ultrasound-Targeted Delivery of Gene-Bearing Nanocarriers

Project Name: Minimally-Invasive Therapy for Parkinson’s Disease Achieved by the Focused Ultrasound-Targeted Delivery of Gene-Bearing Nanocarriers
Status: Complete
Primary Site: Johns Hopkins University School of Medicine
Sponsor Organization: Focused Ultrasound Foundation
Funded Date: May 15, 2019
Funding Period: May 15, 2019 - Apr 30, 2024
Funding Program: Brain Program
Category: Pre-Clinical
Amount Awarded: $2,560,000.00

Abstract

We propose here the development of a Parkinson’s disease treatment approach that entails delivering non-viral neurotrophic gene-bearing nanocarriers to the brain following their intravenous injection into the bloodstream. In Aim 1, so-called “brain-penetrating nanoparticles” will be designed to exhibit uniform, long-lasting, and effective neurotrophic gene delivery. In Aim 2, we will test efficacy of the approach in a small animal model of neurodegeneration. This will entail direct examination of the density of healthy neurons to determine whether neurodegeneration has been slowed or halted, measurement of local levels of dopamine, and assessments of improvements in motor function.

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