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Discovery & Development Drug Discovery, Advanced Medicine

Alleviating the Pressure

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.

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  1. C Chu et al., “Gene Therapy for Glaucoma by Ciliary Body Aquaporin 1 Disruption Using CRISPR-Cas9”, Molecular Therapy, 28, 820, (2020).

About the Author

Maryam Mahdi

Associate Editor

After finishing my degree, I envisioned a career in science communications. However, life took an unexpected turn and I ended up teaching abroad. Though the experience was amazing and I learned a great deal from it, I jumped at the opportunity to work for Texere. I'm excited to see where this new journey takes me!

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