Preclinical studies of Mediphage Bioceuticals ministring DNA gene delivery system for the treatment of Dravet Syndrome

This research project will advance the development of a possible treatment for Dravet Syndrome, a rare form of childhood epilepsy caused by a genetic mutation. In Dravet syndrome, an important protein responsible for sending signals along the nerve fibers of the brain does not function, which results in overactivity and triggers debilitating and recurring seizures. MBI’s gene therapy platform (DNA ministrings) is being developed into a precision medicine that would replace the dysfunctional protein with a functional version. This project will assess the potential safety and effectiveness of this novel gene therapy using advanced human induced pluripotent stem cells. This project will advance the mission of the stem cell network by simultaneously developing new stem cell models and a novel potentially life-saving therapy for an uncurable disease.

Faculty Supervisor:

Michael Beazely

Student:

Partner:

Stem Cell Network;Mediphage Bioceuticals Inc

Discipline:

Life Sciences

Sector:

Professional, scientific and technical services

University:

University of Waterloo

Program: