Ms. Zeinab Mohanna’s research will test the hypothesis that CRISPR/Cas9-mediated genome editing can increase the expression of PAX6 protein, improve the function of the neural and other tissues of the eye, and ultimately rescue the mutant phenotype in the mouse model of aniridia. Rare genetic diseases create a burden on affected individuals, their families, and community, and therefore can be regarded as major public health issue. In the case of aniridia, which is a disease with childhood onset, patients encounter extra-burden as life-long care, surveillance, and management of the disease is required. Tackling this disease with a CRISPR-based gene therapy would be a novel and long-lasting approach, which can cut Canadian healthcare cost spent on managing long-term complications of aniridia.
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Children’s and Women’s Health Centre of British Columbia
