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Genome editing and glaucoma

Genome editing treats glaucoma in mouse model. Image courtesy of iStockphoto/Natali_Mis.

Mutations in the myocilin gene (MYOC) can lead to the misfolding and toxic buildup of the encoded protein in the trabecular meshwork (TM), a tissue that regulates pressure inside the eyes, triggering the death of TM cells, raising intraocular pressure, and leading to glaucoma. Ankur Jain et al. (pp. 11199–11204) used CRISPR-Cas9–mediated genome editing to disrupt the production of mutant myocilin in mouse and human TM cells, as well as in a mouse model of primary open glaucoma. Cells in which mutant MYOC was disrupted exhibited reduced levels of both myocilin and protein biomarkers of TM cell stress. Intravitreal injection of an adenovirus-based CRISPR construct that …

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