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TL;DR
This preclinical study developed a novel gene therapy vector (scAAV2-Trx2-C3) combining a RhoA inhibitor and an antioxidant. In a mouse model of glaucoma, it lowered intraocular pressure and prevented retinal ganglion cell death.
International Journal of Molecular Sciences · 11 authors, 4 centres
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This preclinical study developed a novel gene therapy vector (scAAV2-Trx2-C3) combining a RhoA inhibitor and an antioxidant. In a mouse model of glaucoma, it lowered intraocular pressure and prevented retinal ganglion cell death.
The study developed a self-complementary AAV2 vector expressing a fusion of thioredoxin 2 (Trx2) and C3 transferase (C3) to target glaucoma. In vitro, the vector reduced active RhoA, increased phosphor-cofilin, and inhibited oxidative stress and fibrotic markers in dexamethasone (DEX)-treated HeLa cells. In a DEX-induced mouse glaucoma model, intracameral delivery of scAAV2-Trx2-C3 significantly lowered intraocular pressure (IOP) for up to 4 weeks and protected retinal ganglion cells (RGCs) from apoptotic death, as shown by TUNEL and NeuN staining. It also decreased fibronectin and alpha-SMA expression in eye tissues. The findings suggest potential for a dual-action gene therapy.