Rescue of Endoplasmic Reticulum Associated Degradation of F508CFTR Protein in Cystic Fibrosis by Stop Transfer Sequence Containing Verotoxin

The intern will generate DNA sequences to make modified verotoxin proteins that will effectively disrupt the breakdown of a mutant protein (the CFTR protein) that is found in patients with cystic fibrosis. The mutant CFTR protein has normal function but is degraded in cystic fibrosis individuals because of the mutation. The modified verotoxin will reduce the breakdown of the CFTR protein, and hence increase expression of the mutant CFTR protein and subsequent restoration of normal function of cells expressing CFTR protein. This will enhance the quality of life for cystic fibrosis patients, and could potentially lead to treatment of this disease. LISI Therapeutics will benefit by increasing their license portfolio to include the use of the modified verotoxins as a treatment for patients with cystic fibrosis.

Faculty Supervisor:

Dr. Chetan Tailor

Student:

Abigail Fernandes

Partner:

LISI Therapeutics

Discipline:

Biology

Sector:

Pharmaceuticals

University:

University of Toronto