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CRISPR/Cas9-Mediated Gene Therapy to Correct Aniridia in a Mouse Model

Project Overview

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.

Principal Investigator

Seyedeh Zeinab Mohanna , BCCHR

Partners and Donors

Children’s and Women’s Health Centre of British Columbia

Project Complete

CRISPR/Cas9-Mediated Gene Therapy to Correct Aniridia in a Mouse Model

  • Program Type

    Capacity building grants

  • Area of research

    Central Nervous System

  • Disease Area

    Other

  • Competition

    Brain Canada - Kids Brain Health Network Training Awards

  • Province

    British Columbia

  • Start Date

    2018

  • Total Grant Amount

    $60,000

  • Health Canada Contribution

    $30,000