University of Guelph researchers hope to develop better treatments for cystic fibrosis (CF) and improve patients’ lives through new awards from Cystic Fibrosis Canada.
Pathobiology professor Sarah Wootton has received $157,674 to study a method to permanently treat the disease. Amber Park, a post-doc researcher in the Department of Molecular and Cellular Biology, will use a two-year, $70,000 fellowship to study biofilms made by antibiotic-resistant pathogens in CF infection.
Wootton’s new award brings Cystic Fibrosis Canada’s total investment in her work to nearly $300,000. She hopes to directly address the cause of CF disease by developing a viral gene therapy vector to permanently deliver therapeutic genes to the lung.
“I feel a strong sense of commitment to the CF community to achieve our research objectives and develop gene therapy vectors that may one day be used to ‘functionally cure’ individuals with CF,” said Wootton, who joined U of G in 2007 as a professor in the Ontario Veterinary College.
“We are grateful to Cystic Fibrosis Canada for their continued support of our research, without which this work would not be possible.”
Cystic fibrosis is the most common fatal genetic disease affecting Canadian children and young adults. Nearly 60 per cent of all Canadians with CF are adults. There is no cure.
Build-up of thick mucus in the lungs causes severe respiratory problems.
The disease occurs when a child inherits a defective copy of the pertinent gene from each parent. One in 25 Canadians is a CF carrier, but many people are unaware of their status.
“The good thing, from a genetic standpoint, is that because we know mutations in one gene are responsible for the disease, we can target that gene and replace it with a functional copy in hopes of reversing the lung disease,” Wootton said.
Using genetic engineering, she is manipulating a virus to carry a normal version of the CF gene into lung cells. Studies suggest that restoring 10 to 20 per cent of the protein produced by the CF gene is enough to mitigate disease symptom, she said.
“Viruses have evolved to deliver genetic information to cells very effectively,” Wootton said. “We are taking advantage of the natural ability of a virus to target the lung and incorporating novel genome editing technologies into these vectors with the aim of permanently delivering functional genes to the lungs.”
The particular virus is safe to use in humans as it “cannot replicate in mammalian cells. You use it as a vehicle to deliver the DNA, and once inside the cell, it expresses the therapeutic protein but cannot produce more of itself.”
The virus also has a large genome, she added. “You can pack a lot of genetic information into it.”
Park works with Prof. Cezar Khursigara, Department of Molecular and Cellular Biology, who previously received more than $200,000 from Cystic Fibrosis Canada.
They plan to learn more about how biofilms made by Pseudomonas aeruginosa help these bacteria resist antibiotics. P. aeruginosa is one of the most harmful pathogens in CF infections.
The researchers believe their work can help improve treatment and, ultimately, improve the quality of life of CF patients.
Park said: “I am deeply honoured to have the importance of my work recognized by the CF research community, and I am excited to further develop the Khursigara lab’s already successful research program.”
She was named Cystic Fibrosis Canada’s 2015-16 Kin Canada Fellow as the top-ranked post-doctoral researcher in the field.