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Novel strategies for concurrent gene delivery to central and peripheral organs

Carlsbergfondets internationaliseringsstipendier

What

My research focuses on gene therapy, which is based on delivery of therapeutic genes to the relevant tissue and organs in the body. Therapeutic genes can correct inherited diseases or ameliorate pathologies that develop later in life. Gene therapy can be given as a one-time treatment to provide long-lasting benefits, which is especially promising for diseases that can persist for decades or even for life. For therapeutic genes to exert their beneficial effects they must be delivered to the affected part of the body. This delivery is especially challenging in diseases affecting multiple organs, including the brain. The goal of my project is to develop novel strategies to safely and efficiently achieve simultaneous delivery of genes to multiple organs and several parts of the brain.

Why

Gene delivery to multiple organs, and especially to several parts of the brain, remains an unmet need for many devastating disorders. My project will provide a platform to advance scientific fields related to gene therapy and propel forward the possibilities to deliver treatments across the human body and for a broad range of diseases. This will allow for the development of long-lasting treatments for diseases that currently have no cure. The gene delivery can be modified for specific needs for the individual treatments and be tailored for widespread gene delivery to organs, including the brain, or focus on efficient delivery to multiple or single brain regions.

How

Gene delivery to organs, except the brain, are easily achieved by a simple intravenous injection of gene carriers. A major obstacle for gene delivery to the brain is the blood-brain-barrier, which only allows few substances to pass from the blood into the brain. I will overcome this barrier by using transcranial focused ultrasound and microbubbles injected in the blood. The interaction between the ultrasound waves and the microbubbles circulating in the blood vessels of the brain causes a temporary increase in the ability of gene carriers to travel from the blood into the brain. I will conduct this project at Sunnybrook Research Institute in Toronto, Canada. Sunnybrook is recognized as 1 of only 8 Centre of Excellence in Focused Ultrasound in the world.

SSR

The ability to deliver genes to multiple organs and several brain regions with a single treatment can lead to therapeutic efficacy for a broad range of disorders. Gene therapy can provide lasting benefits for people suffering from inherited and acquired diseases that require life-long treatments. For example, my research includes delivery strategies that can lead to effective treatments for people living with neurodegenerative diseases such as Alzheimer's disease, for which there is no cure. The methods developed within the scope of my project can be combined with novel and pre-existing therapeutics; which we are developing and investigating with industry partners, respectively. Promising compounds can only reach therapeutic efficacy provided that sufficient delivery is achieved.