Alleviating the Pressure
Gene therapy shows promise for glaucoma in a mouse model
Researchers from the University of Bristol, UK, have used gene therapy to tackle glaucoma – a common cause of blindness and a disease with no cure. In an attempt to lower intraocular pressure (IOP) – a common mode of intervention – the researchers used a single intravitreal injection to deliver gene therapy that reduced the production of aqueous humor in the eye (by disrupting Aquaporin 1). In turn, the lowered pressure prevented damage and, importantly, preserved nerve cells in the induced mouse model of ocular hypertension (1).
“Though most patients use daily eye drops, these have side effects,” explains Colin Chu, a research fellow at the university. “Glaucoma surgery is also available but it requires skilled surgeons, intensive follow up, and often fails over time.”
According to the researchers, the approach, which combines CRISPR-Cas9 with viral gene vector technology, has the potential to provide long-lasting IOP reduction with a single injection – and that could help alleviate the treatment burden for patients and clinicians alike.
- C Chu et al., “Gene Therapy for Glaucoma by Ciliary Body Aquaporin 1 Disruption Using CRISPR-Cas9”, Molecular Therapy, 28, 820, (2020).
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