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Cystic Fibrosis Research Aims to Permanently Treat the Disease

Ontario Veterinary College Prof. Sarah Wootton is developing a viral gene therapy vector to permanently deliver therapeutic genes to the lung

Jehangir Saleh used his philosophy studies at U of G to help him understand and cope with cystic fibrosis (CF), a disease that took his life in 2013. Although he was unable to complete his master’s degree due to his illness, his writing lives on, giving hope to those with CF and other chronic diseases.

He often compared life with CF to mopping a floor that always gets dirty again. His treatment, which kept him in St. Michael’s Hospital in Toronto for up to six months at a time, involved several hours of drug therapy per day to help clear his lungs of mucous, only to be repeated the next day as his lungs filled up with mucous again.

“This aspect of CF life was emotionally difficult for me,” he wrote on his blog, Philosophy of Illness. “For someone who spent his life being an overachiever, who felt like, with enough hard work, I could make things go my way, here was one thing that, no matter what I did, I couldn’t control.”

Jehangir’s younger sister, Jasmine, remembers him as a mentor who helped her put her life in perspective. She says a 10-minute conversation with him made an impression on her friends, who often told her, “Your brother really left me thinking about my life.”

There’s hope for CF patients in the form of research that could lead to more effective treatments – and possibly a cure. Pathobiology professor Sarah Wootton has received almost $300,000 in funding from Cystic Fibrosis Canada to study gene therapy that aims to replace the defective gene that causes CF with a functional one.

Cystic fibrosis is caused by the dysfunction of a single gene, the cystic fibrosis transmembrane conductance regulator (CFTR); 90 per cent of CF patients have a single mutation that causes the CFTR protein to malfunction.

“What we do in our lab is modify viruses to use as gene therapy vectors,” says Wootton. “We are engineering baculovirus vectors to permanently correct the CFTR gene, and we’re doing this using state-of-the-art site-specific genome editing techniques.”

Read the entire article on the University of Guelph website.