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Qin Liu, Ph.D., M.D.

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Available: 07/05/17, Expires: 05/31/19

Inherited retinal degenerations (IRDs) are important causes of vision loss that affect people of all ages. These disorders cause vision loss via dysfunction and death of the photoreceptor and retinal pigment epithelial cells of the retina. Gene augmentation therapy has been shown to be beneficial for patients with specific genetic forms of IRDs to date, but IRDs are genetically heterogeneous, and gene therapy may not be possible for all of the different genetic forms of disease, such as those caused by mutations in large genes, or by dominant gain-of-function mutations. This project is to test the use of CRISPR/Cas9 – mediated genome editing to treat several genetic forms of retinal degeneration that are not amenable to standard gene augmentation therapy approaches. The role of the defined student will be to learn and apply skills in genetics, molecular and cellular biologic, and CRISPR/Cas9 genome editing for treating one of the dominant forms of IRD in patient-derived iPS cells and gene targeted mouse models.

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