In vitro development of LNP-mRNA as a treatment for neurological dysfunction in Gaucher Disease

Brain disorders affect 10% of Canadians, however few treatment options exist for these devastating
diseases. We have extensive understanding of what causes many of them, but current medical technology
has yet to translate this knowledge into effective treatments. 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 to treat brain disorders. Using a technology similar to the COVID vaccine that combines
lipid nanoparticles (LNP) and messenger RNA (mRNA), we can transform brain cells into protein factories
that continually produce these therapeutics proteins over days to weeks. We have proof of concept data in
mice demonstrating this strategy is tunable and achieves long lasting brain-wide biodistribution of desired
proteins, requiring fewer infusions compared to existing treatments.

Faculty Supervisor:

Brian MacVicar

Student:

Partner:

CereCura Nanotherapeutics

Discipline:

Life Sciences

Sector:

Professional, scientific and technical services

University:

The University of British Columbia

Program: