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Dr. Simpson – Using Gene Therapy To Treat Congenital Blindness

Scientific Title: Intravenous rAAV MiniPromoter-PAX6 gene therapy for the congenital blindness aniridia

Lead Investigator: Dr. Elizabeth M Simpson
Institution: University of British Columbia
Granted: $296,747 over 2 years

Aniridia is an eye disorder where the iris, the coloured part of the eye, is partially or completely absent. Individuals with aniridia usually have low vision from birth and develop glaucoma and cataracts, which can lead to blindness. Most cases of aniridia are caused by a mutation in the PAX6 gene. Dr. Simpson and her team will test gene therapy as a new treatment option for this gene. The team will study the effectiveness of PAX6 gene therapy in an animal model of aniridia. This is an important step before this treatment can be considered for human trials.

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