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This project will test how well different DNA-based therapies work in the brain and body using a mouse model. Mice will receive either a high or low dose of the therapy, and their response will be tracked through imaging, blood tests, and tissue analysis. The goal is to measure how much of the DNA reaches target areas and whether it produces the intended protein. Results will be analyzed statistically and shared with project partners. This work supports the development of safer, non-viral gene therapies and could lead to future treatments for neurological and systemic conditions.
This project supports the partner organizations R&D goals by optimizing how novel DNA therapies are delivered, aiming to improve treatments for rare and brain-related genetic disorders. It will generate key data to guide the design and delivery of safer, redosable gene therapies. These results will help advance msDNA technology toward clinical use and address major gaps in current treatment options, boosting the partners productivity and innovation pipeline.
Jagdeep Walia
Stem Cell Network;Mediphage Bioceuticals Inc
Life Sciences
Professional, scientific and technical services
Queen's University
Accelerate
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