Turn off aquaporin

Confirmed: One shot of CRISPR can cure glaucoma

Sergey Kolenov, Hi-tech+

Experiments on mice and human tissues have confirmed that inactivation of a single gene using CRISPR can reduce intraocular pressure, which would otherwise lead to blindness. Clinical trials with human participation are next in line. If they are successful, 64 million people suffering from glaucoma will be able to queue up for a therapeutic injection.

Glaucoma is considered the main cause of irreversible blindness. In patients suffering from this disease, fluid accumulates in the front of the eye, resulting in increased intraocular pressure and damaged optic nerves. Modern methods of glaucoma treatment include eye drops, laser therapy and surgery, but each of them has limitations and disadvantages.

A new approach to the fight against this disease was presented by specialists from the University of Bristol, whose work is described in the press release Study shows glaucoma could be successfully treated with gene therapy. It is based on the use of CRISPR gene therapy.

Article by Wui et al. Gene therapy for glaucoma by ciliary body Aquaporin 1 disruption using CRISPR-Cas9 is published in the journal Molecular Therapy – VM.

Scientists suggest using CRISPR to inactivate the Aquaporin 1 gene in the ciliary body of the eye. This structure supports the lens and produces a watery liquid that fills the eye chamber. By disabling Aquaporin 1, you can reduce the production of this fluid and thereby reduce intraocular pressure.

Experiments on mice and human tissues have already confirmed the efficiency of the technique. In the near future, the team plans to switch to clinical trials in humans. If they are successful, 64 million people suffering from glaucoma worldwide will have a chance of healing with a single injection.

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