Blair Leavitt

MD

Investigator, BC Children's Hospital
Senior Scientist, CMMT

My laboratory develops new treatments for neurodevelopmental disorders such as pyridoxine dependent epilepsy and neurodegenerative diseases such as Huntington's disease. We do this by first developing transgenic mouse models of specific human diseases, and then by testing new disease treatments in these mouse models. The YAC128 mouse model of Huntington's disease accurately replicates the changes seen in humans with this disease, and is a critical tool that permits my laboratory to evaluate new treatment strategies in mice prior to undertaking large-scale clinical trials in human patients. My work was initially focused on transgenic mouse models of Huntington's disease, but has now expanded to utilize the same standardized approach to investigate new therapeutics in mouse models of other devastating neurodevelopmental and neurodegenerative disorders, such as Frontotemporal dementia and Fragile X syndrome.