Identifying potentiators for improved adeno associated virus based gene therapy for the central nervous system

Gene therapies are revolutionizing medicine, providing new therapeutics for diseases that previously were untreatable. In the central nervous system in particular, gene therapies hold great promise for saving lives and improving the quality of life of children and aged patients. The most common form of gene therapy that is used to treat patients involves the use of a virus deliver the gene of interest. However, getting enough into the cells of the patients is difficult, and represent a significant challenge to creating more life saving gene therapies. Here we are testing how drugs, called vector potentiators (VEPOs™), can be used to improve viral delivery of genes to the central nervous system, providing a platform for the further development of gene therapies.

Faculty Supervisor:

Cindi M Morshead

Student:

Partner:

Stem Cell Network;Virano

Discipline:

Life Sciences

Sector:

Professional, scientific and technical services

University:

University of Toronto

Program: