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CRISPR used to prevent glaucoma in mice

The breakthrough came following 24 years of collaborative study and was led by researchers from the University of Iowa (UI), who eliminated the mutated myocilin protein from a mouse model of human glaucoma and cultured human cells. Mutations in myocilin are implicated in juvenile and adult-onset primary open-angle glaucoma.{{quote-A:R-W:450-I:2-Q: As scientists, we don’t want to just discover a diseased gene, we want to understand what the gene does and, in this case, have a better understanding of glaucoma so that it can be more effectively treated. -WHO:Dr Val Sheffield, study author}}Using CRISPR-Cas9, the team found that roving the mutated protein resulted in lowered intraocular pressure, thus preventing glaucomatous damage.“As scientists, we don’t want to just discover a diseased gene, we want to understand what the gene does and, in this case, have a better understanding of glaucoma so that it can be more effectively treated. No one knows what this gene does, except that its mutant form causes glaucoma,” study author Dr Val Sheffield said.Myocilin is found in the trabecular network responsible for regulating intraocular pressure, and a mutated gene of the protein can cause pressure to build up.The team found that disrupting the mutated gene stopped the production of the mutant protein in the mouse model, thus inhibiting intraocular pressure and eventually preventing the human form of glaucoma from occurring.The same gene editing technology stopped myocilin expression in perfusion cultured human eyes, which the researchers suggest, could translate into new and unique therapies in treating myocilin glaucoma.The findings were the result of collaborative study between scientists from the UI Carver College of Medicine, Wynn Institute for Vision Research, North Texas Eye Research Institute from the University of Northern Texas Health Science Centre and McGovern Institute for Brain Research at the Massachusetts Institute of Technology.The study was published in Proceedings of the National Acady of Science.