Innovative Gene Therapy Lowers Eye Pressure in Mice as a Reversible Alternative to Glaucoma Drops

Recent advances in gene editing have demonstrated promising results in managing intraocular pressure (IOP), a key factor in glaucoma development. Researchers utilized the CRISPR-based effectors, specifically Cas13d, to target and downregulate two genes—AQP1 and CA2—that are integral to aqueous humor production within the ciliary bodies of mouse eyes. This innovative approach led to a significant reduction in eye pressure: treated mice experienced a decrease of approximately 2.5 mmHg and 1.7 mmHg in IOP, compared to control groups. Importantly, this method does not permanently alter the DNA, making it a reversible and adjustable treatment option. The study, published in PNAS Nexus, indicates that such gene therapy could be adapted for human use, requiring only monthly or less frequent administrations. While further research is necessary to optimize timing, dosage, and delivery methods, this technique offers a potential paradigm shift in glaucoma management, reducing reliance on daily eye drops that can cause adverse effects and are often poorly adhered to by patients.