Researchers successfully demonstrate gene therapy for glaucoma in the lab

Lead researcher Dr Colin Chu, from University of Bristol said: “The benefit of this new treatment is that with one simple injection we could potentially reduce someone’s eye pressure for a long time. In theory because it permanently edits the genetic code, a single treatment might be all you need.” 
A man with black hair in a lab smiling at the camera.
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What was the aim of the research?

A Fight for Sight funded researcher from the University of Bristol, Dr Colin Chu, has used gene editing techniques to successfully demonstrate a gene therapy for glaucoma in the lab. The results of this study were published in Molecular Therapy in March 2020.

Why was the research needed?

Glaucoma is the second leading cause of blindness for which there is currently no cure. This research could form a new treatment approach for glaucoma. With current treatments, patients who use eyedrops can experience side effects, have to use them every day and they don’t always work sufficiently. There’s also glaucoma surgery, but there are risks, extensive follow-up is needed and it can fail over time.

What method did researchers use?

In a lab setting, the researcher used a gene-editing technique called CRISPR to alter genetic information when transferred into eye cells using a harmless virus.

By switching off a gene called aquaporin 1, researchers found that the gene therapy prevented the build-up of fluid – and therefore the increase in eye pressure that results in damage to the optic nerve in glaucoma. Aquaporin 1 is known to be associated with the production of eye fluid in glaucoma but until now there has been no therapy acting on this gene to treat glaucoma.

What impact will it have?

The findings are incredibly promising, particularly for those who do not experience a reduction in eye pressure despite the current treatment.

Researchers hope that with one simple injection,a person’s eye pressure could potentially be reduced for a long time. In theory because it permanently edits the genetic code, a single treatment might be all that is needed.

Dr Chu hopes the potential treatment can be taken to a clinical trial in the next few years. If taken forward, a new gene therapy could be available in the next decade. 

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