Gene Therapy for Cystic Fibrosis: Going Viral.

Gelinas JF

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Oxbridge Canadian Conference, Canada House, London, 2013

Gene therapy offers the potential to treat a range of genetic conditions including lung disease associated with Cystic Fibrosis (CF). CF is the most common lethal inherited disease affecting Canadian children and young adults. One in every 3,600 children born in Canada has CF, with a median life expectancy of only ~36 years.

Typically, CF individuals suffer from repeated bacterial infections of the airways eventually leading to respiratory failure. To date, no treatment that targets directly the genetic defect is available, despite its identification in 1989. Our aim is to utilise a virus specifically adapted to enter airway cells to transfer the correct gene to CF patients.

This research will be translated to the clinic in order to prevent decline in lung function and early death. Development of this new viral technology is also an exciting opportunity to treat other lung diseases such as chronic obstructive pulmonary disease (COPD) and emphysema.