Related Experiment Video
Updated: Jan 7, 2026

Drug Repurposing Hypothesis Generation Using the "RE:fine Drugs" System
Published on: December 11, 2016
Drug Development
Malik White1,2, Rikke Han Kofoed3,4,5, Chinaza Lilian Dibia1,2
1University of Toronto, Toronto, ON, Canada.
Background:
Recombinant adeno-associated viruses (AAVs) hold promise for brain disorders but often require intracranial injection, limiting therapeutic reach and being invasive. Intravenous (i.v.) AAV delivery is restricted by the blood-brain barrier (BBB), reducing efficiency. Recent AAV variants can cross the BBB, enabling widespread brain transduction. MRI-guided focused ultrasound (FUS) transiently modulates BBB permeability, enhancing gene delivery in small or large brain regions targeted.
Hypothesis:
We hypothesize that combining IV BBB-penetrating AAVs with FUS will optimize gene delivery, enabling both widespread and region-specific transduction for neurodegenerative diseases.
Methods:
Three-month-old C57BL/6 mice received i.v. AAV-PHP.V1.CAG.TdTomato (V1) and AAV9.CAG.EYFP, followed by FUS targeting various brain regions. Immunohistochemistry and confocal microscopy were done 3 weeks post-delivery.
Results:
Our findings demonstrate that FUS enhances targeted gene delivery for AAV9 and V1 while maintaining global expression in the brain for V1, offering a non-invasive strategy for precise (AAV9) or widespread and locally enriched (V1) gene therapy.
Conclusion:
This approach could improve treatments for neurodegenerative disorders such as Alzheimer's and Parkinson's disease.
More Related Videos
08:04In Vitro Three-Dimensional Sprouting Assay of Angiogenesis Using Mouse Embryonic Stem Cells for Vascular Disease Modeling and Drug Testing
Published on: May 11, 2021
05:45Developmental Toxicity Assay Based on Real-Time Monitoring of Fibroblast Growth Factor Signal Disruption in Human Induced Pluripotent Stem Cells
Published on: October 10, 2025
Related Concept Videos
Preclinical Development: Overview
Clinical Trials: Overview
Drug Discovery: Overview
Drug Administration and Therapy Phases: Overview
The pharmaceutical phase focuses on leveraging the physicochemical properties of the drug to design and manufacture an effective product. Variants include orally administered tablets or capsules, topical creams or ointments, and parenteral-delivery solutions or emulsions.
The pharmacokinetic phase...
In Vitro Drug Release Testing: Overview, Development and Validation
Drug Regulation