Using gene therapy to treat congenital blindness
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. Current treatments may slow the progression of aniridia, but there are currently no cures or treatments to maintain vision long term. Dr. Elizabeth M Simpson is looking to gene therapy for a possible solution. The majority of aniridia cases are caused by a mutation in the PAX6 gene, which plays an important role in eye development. Dr. Simpson’s team will use gene therapy to put a functioning copy of the PAX6 gene back into the eye of mice who have a mutation in PAX6 and assess whether this improves or even restores vision. This project is an important step before we can consider giving this treatment to patients. Dr. Simpson’s research is taking a big leap forward in our search for a cure for aniridia and will also provide important information to help scientists working on gene therapies for other eye diseases.
Full grant release details here: https://www.fightingblindness.ca/news/2019-research-grant-winners