Non-viral, immune-modulatory nanoparticles for delivery of CRISPR/Cas9 as a treatment intervention for Duchenne Muscular Dystrophy

2019

Dr. Anthony Gramolini
University of Toronto, Toronto, Ontario

Lead investigator

Dr. Anthony Gramolini

Dr. Anthony Gramolini
University of Toronto
Toronto, Ontario

Collaborators & Co-Investigators

  • Ronald Cohn, MD

Research Sites & Affiliations

  • The Hospital for Sick Children, Toronto, Ontario

Funding partners: Jesse’s Journey

Budget: $300,000

Disorders: Duchenne/Becker Muscular Dystrophy

Research Areas: Discover Novel Treatments & Therapies

Abstract: 

We are developing non-viral vehicles for delivery of genome editing machinery with specific emphasis on targeting skeletal muscle cells associated with Duchenne muscular dystrophy (DMD). Clustered regularly interspaced palindromic repeats (CRISPR)-CRISPR associated protein 9 (Cas9) is a powerful new gene editing tool. Our objective focuses on generating novel degradable and biocompatible nanoparticles (BNPs), using our U of T patented polyurethane technology. These carriers address limitations with current CRISPR/Cas9 delivery platforms, specifically eliminating use of immune reactive virus; enables co-delivery of a specific targeting tool; and reduces potential off-target tissue damage; . Studies will evaluate the therapeutic corrective capacity of nanoparticles in a DMD mouse model, and establish a technology to enable novel therapies for DMD patients in Canada and abroad.


Impact: