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Gene Therapy in Mice


We create mouse models of human disease by gene editing (see CRISPR Gene Editing Project). We can then use these mouse models to test treatments for a particular disease by delivering the gene therapy, correction of a genetic mutation or providing an extra, healthy copy of a gene allele, into the mouse eye (See Microsurgery Page). We can examine these treated mice with our live imaging technology to monitor their vision over their lifespan. If the treatment is successful, this acts as a preclinical test for future gene therapy human clinical trials to treat eye disease.