Dr. Balasankara Kaipa from Gavin Herbert Eye Institute awarded $90,000 Knights Templar Eye Foundation Grant for Juvenile Glaucoma Therapy Research

Dr. Balasankara Kaipa from the Gavin Herbert Eye Institute, Center for Translational Vision Research, Irvine, California was awarded a $90,000 grant entitled: Lipid Nanoparticle-Mediated Antisense Oligonucleotide Therapy for Juvenile Glaucoma. Mutations in the myocilin gene cause a severe form of Juvenile glaucoma.

Young children with this mutation often develop high intraocular pressure and present clinically with severe form of glaucoma. Juvenile glaucoma with this mutation is often difficult to treat since current medication do not directly target mutated gene. There is a critical need to develop therapeutic strategies targeting myocilin. Dr. Kaipa’s previous studies have shown that mutant myocilin causes protein misfolding leading to its abnormal accumulation in trabecular meshwork cells, which regulate intraocular pressure and reducing myocilin in trabecular meshwork genetically rescued myocilin glaucoma in mice. Here, Dr. Kaipa proposes to target myocilin using lipid nanoparticles mediated delivery of Antisense oligonucleotide (ASO) that bind to myocilin and inhibit protein synthesis, thereby reducing its accumulation. The preliminary studies show that they can deliver genetic materials to trabecular meshwork and efficiently reduce mutant myocilin using ASOs in cultured cells. They will test whether these lipid nanoparticles reduce mutant protein and rescue a mouse model of glaucoma caused by these mutations. The successful completion of these studies will identify novel therapeutic targets for myocilin-associated glaucoma.