Cas13d Gene Editing Shows Promise for Glaucoma Treatment

Gene editing may offer a new approach to treating glaucoma. Glaucoma is a leading cause of blindness and is associated with elevated pressure inside the eye. Patients are often prescribed daily eye drops to reduce this pressure. However, these medications can cause side effects such as kidney stones, metabolic acidosis, and bradycardia. In addition, many patients do not use the drops consistently.

Yang Sun and colleagues used CRISPR gene editing to target two genes involved in producing aqueous humor, the fluid inside the eye. They used Cas13d, a CRISPR effector that modifies RNA, to target the mRNAs for aquaporin 1 (AQP1) and carbonic anhydrase type 2 (CA2) in the ciliary body of mouse eyes.

Mice treated with Cas13d showed a significant reduction in intraocular pressure compared to untreated controls. This effect was observed in both normal mice and mouse models of glaucoma.

The procedure does not make permanent changes to the DNA of eye cells. This means the treatment is reversible and could be adjusted over time.

The authors note that further research is needed to develop a human therapy. Key factors include identifying the appropriate timing, dosage, and a noninvasive method of delivery. They suggest that, if effective, the treatment might be needed only once a month or less.