Promising gene editing techniques have been used to treat glaucoma in the lab, it has been announced.
Dr Colin Chu, who is leading the research at University of Bristol, UK, used CRISPR to alter genetic information when transferring into eye cells using a harmless virus.
When Dr Chu and his colleagues switched off aquaporin 1, which is associated with the production of eye fluid in glaucoma, they found that the gene therapy prevented the build-up of the fluid.
It is hoped that the findings, which are published in the latest edition of Molecular Therapy, could lead to clinical trial in the next few years, with a possible gene therapy being available in the next decade.
“With current treatments, patients who use eyedrops can experience side effects, have to use them every day and they don’t always work sufficiently,” said Dr Chu.
“There’s also glaucoma surgery, but there are risks, extensive follow-up is needed and it can fail over time. 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.”
Dr Rubina Ahmed, head of research at Fight for Sight, which funded the study, described the findings as “incredibly promising”, particularly for people who do not experience a reduction in eye pressure despite the current treatment.
“Dr Chu’s work represents a significant breakthrough for eye research and shows the importance of science to find new treatments for the prevailing causes of sight loss,” she added.
In this study, it was demonstrated that a gene therapy approach reduces eye pressure by disrupting fluid production in the ciliary body following a single injection into the centre of the eye.
When tested in mice with induced glaucoma in the lab, using CRISPR reduced the eye pressure without adverse effect.
Gene Therapy for Glaucoma by Ciliary Body Aquaporin 1 Disruption Using CRISPR-Cas9. Molecular Therapy 4 March 2020
https://www.cell.com/molecular-therapy-family/molecular-therapy/fulltext/S1525-0016(20)30007-1
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