Synthesis and characterization of various nanocarriers and ministring DNA product for ocular gene delivery

Gene therapy has the potential to effectively treat a wide range of ocular disorders. However, the introduction of foreign therapeutic genes to cells is incredibly challenging. Compared to viral vectors, non-viral vectors (NVV) are far less immunogenic and less toxic. However, NVV are less efficient than viral vectors at delivering therapeutic genes because they must overcome numerous cellular barriers. An appropriate design of the components of the NVV will enhance the properties of the vector to improve delivery into the cell. Our group is working to optimize various nanocarriers and develop a DNA NVV that exploits Mediphage’s proprietary miniaturized DNA construct, called ministring DNA (msDNA). msDNA vectors confer higher transfection efficiency than traditional plasmid DNA strategies, and their smaller size allows for the delivery of higher copy numbers of therapeutic genes. We are confident that our strategy will lead to the development of an effective NVV for ocular gene delivery.

Faculty Supervisor:

Shawn Wettig

Student:

Gurmeet Lall

Partner:

Mediphage Bioceuticals Inc

Discipline:

Pharmacy / Pharmacology

Sector:

Manufacturing

University:

University of Waterloo