MRGFUS GENE DELIVERY FOR NEUROPROTECTION IN PARKINSON'S DISEASE

Project Name: MRGFUS GENE DELIVERY FOR NEUROPROTECTION IN PARKINSON'S DISEASE
Status: Active
Primary Site: Weill Cornell Medical Center
Sponsor Organization: Focused Ultrasound Foundation
Funded Date: Jan 18, 2019
Funding Program: Brain Program
Funding Period: Jan 18, 2019 - Jan 18, 2020
Amount Awarded: $100,000.00
Category: Parkinson’s Disease

Abstract

GENE THERAPY HOLDS GREAT PROMISE FOR TREATING MANY BRAIN DISEASES. MR GUIDED FOCUSED ULTRASOUND (MRGFUS) PERMITS NONINVASIVE DELIVERY OF GENE THERAPY AGENTS TO SPECIFIC BRAIN REGIONS, YET SPECIFIC TYPES OF NEURONS WITHIN A GIVEN AREA SERVE DIFFERENT FUNCTIONS. WE PROPOSE A NEW TECHNOLOGY USING MRGFUS TO TARGET SPECIFIC NEURONS WHICH MAKE UP DEFINED BRAIN CIRCUITS. WE WILL TEST THE ABILITY OF THIS NEW APPROACH TO DELIVER A POWERFUL GROWTH FACTOR GENE TO PROTECT THE AT RISK DOPAMINE NEURONS IN A PARKINSON'S DISEASE MODEL. SUCCESS OF THIS COULD TRANSLATE INTO MORE EFFECTIVE AND SAFE THERAPIES FOR VARIOUS NEUROLOGICAL AND PSYCHIATRIC DISORDERS.

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