Researchers have concluded that gene editing could become a feasible therapeutic approach for glaucoma, after their study demonstrated that RNA-based gene editing can effectively “knock down” aquaporin 1 (AQP1) and carbonic anhydrase type 2 (CA2) in mouse eyes. 

AQP1 and CA2 are genes associated with the production of aqueous humour, which affects intraocular pressure (IOP). IOP is primarily regulated by the balance between aqueous humour production in the ciliary body of the eye and its outflow in the trabecular meshwork. Using Cas13 RNA editing systems – a revolutionary gene manipulation technique that eliminates the risk of introducing permanent changes to the genome – researchers were able to reduce IOP significantly in both normal mice and those with steroid-induced ocular hypertension.