GLAUCOME: Treat it with a single injection of gene therapy?

This research team from Bristol Medical School presents here a new therapeutic option to treat glaucoma, one of the main causes of blindness in the world: it is a gene therapy, administered by injection and which targets the gene responsible for eye pressure. Work presented in the journal Molecular Therapy that opens up a whole new path, full of hope.

Glaucoma affects more than 64 million people worldwide and is one of the leading causes of irreversible blindness, especially in people over the age of 60. The condition is caused by the accumulation of fluid in the front part of the eye, which increases the pressure inside the eye and gradually damages the optic nerve. Current treatments include eye drops, laser or surgery, treatments that have limits and cause side effects. Lead author Dr. Colin Chu explains that there is currently no cure for glaucoma, which can lead to vision loss if the disease is not diagnosed and treated early enough.

Treat glaucoma with a single injection using gene therapy

A first proof of concept: the researchers bring here in vitro and in vivo a first proof of concept of therapy, on human tissues and in mice “models” of glaucoma. The therapy targets a part of the eye called the ciliary body (see histological section on visual 2), which produces the fluid that maintains pressure in the eye. Using the gene editing technology called CRISPR, researchers inactivate a gene called Aquaporin 1 in the ciliary body, which has reduced eye pressure.

1 injection would be enough: glaucoma could be successfully treated with a single injection using gene therapy, which would improve the treatment options, effectiveness and quality of life of many patients. “We hope to move quickly towards clinical trials. If they are successful, then we would have long-term treatment for glaucoma with a single eye injection, which would improve the quality of life for many patients while saving health care resources. “