Gene Therapy for Glaucoma

Glaucoma
“The major risk factor for glaucoma is elevated intraocular pressure, which is regulated by the balance between the secretion of aqueous humor and the resistance to its flow across the conventional outflow pathway. Here, we [researchers at the Smurfit Institute, Trinity College, Dublin] show that adeno-associated virus 9 (AAV9)–mediated expression of matrix metalloproteinase-3 (MMP-3) can increase outflow in two murine models of glaucoma and in nonhuman primates. We show that long-term AAV9 transduction of the corneal endothelium in the nonhuman primate is safe and well tolerated. Last, MMP-3 increases outflow in donor human eyes. Collectively, our data suggest that glaucoma can be readily treated with gene therapy–based methods, paving the way for deployment in clinical trials.” MORE
Image Credit: Harvard Health