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Study Shows Glaucoma Might Be Successfully Treated with Gene Therapy Using CRISPR-Cas9 Gene Editing to Inactivate Aquaporin 1 Gene in Ciliary Body That Produces Fluid That Maintains Pressure in Eye; Treatment Lowers Intraocular Pressure in Mouse Model

A new study led by scientists at the University of Bristol in the UK has shown that a common eye condition, glaucoma, might be successfully treated with a single injection using gene therapy, which would improve treatment options, effectiveness, and quality of life for many patients. Glaucoma affects over 64 million people worldwide and is a leading cause of irreversible blindness. It is usually caused by fluid building up in the front part of the eye, which increases pressure inside the eye and progressively damages the nerves responsible for sight. Current treatments include either eye drops, laser, or surgery, all of which have limitations and disadvantages. The research team led by academics at the Bristol Medical School: Translational Health Sciences tested a new approach that might provide additional treatment options and benefits. Their findings were published in the March 4,2020 issue of Molecular Therapy. The open-access article is titled “Gene Therapy for Glaucoma by Ciliary Body Aquaporin 1 Disruption Using CRISPR-Cas9.” The researchers designed a gene therapy and demonstrated proof of concept using experimental mouse models of glaucoma and human donor tissue. The treatment targeted part of the eye called the ciliary body, which produces the fluid that maintains pressure within the eye. Using the latest gene-editing technology called CRISPR, a gene called Aquaporin 1 in the ciliary body was inactivated leading to reduced eye pressure. Dr Colin Chu, Visiting Senior Research Fellow in the Bristol Medical School: Translational Health Sciences and corresponding author, said: "Currently there is no cure for glaucoma, which can lead to loss of vision if the disease is not diagnosed and treated early. "We hope to advance towards clinical trials for this new treatment in the near future. If it's successful it could allow a long-term treatment of glaucoma with a single eye injection, which would improve the quality of life for many patients whilst saving the NHS time and money." The academics are currently in discussion with industry partners to support further laboratory work and rapidly advance this new treatment option towards clinical trials.

[Press release] [Molecular Therapy article]