In vivo testing of LNP-mRNA therapeutics in an animal model of Gaucher Disease

Brain disorders affect 10% of Canadians, however few treatment options exist for these devastating diseases. We have extensive understanding of their causes, but translation to treatment has been difficult. For example, many natural proteins have been identified to hold significant therapeutic promise, but getting these proteins to the brain has been a longstanding challenge. Our group has developed a technology that empowers the brain to produce its own therapeutic proteins over days to weeks. Using a technology similar to the COVID vaccine (combining lipid nanoparticles (LNP) and messenger RNA (mRNA)), we propose to transform brain cells into factories for the production of the protein deficient in Gaucher Disease (GD), in order to improve treatment strategies for the neurological manifestations of this disorder and provide proofof- concept for our strategy. By demonstrating the efficacy of this treatment in animal models, we hope to support its development for a multitude of brain disorders.

Faculty Supervisor:

Brian MacVicar

Student:

Partner:

CereCura Nanotherapeutics

Discipline:

Life Sciences

Sector:

Professional, scientific and technical services

University:

The University of British Columbia

Program: