Potential Treatment Of Corneal Vascularization

NU 2018-012

Inventors

Tsutomu Kume

Short Description

A gene therapy approach to diminish corneal vascularization

Background

Normally devoid of blood vessels, the cornea can become vulnerable to vascularization due to bacterial and viral infections, chemical injury, autoimmune conditions, and post-corneal transplantation, which can lead to sight-threatening conditions. The current treatment options are not effective and limited. Specifically, patients with PAX6 mutation develop corneal vascularization and progressively lose their vision over 2-3 decades. This condition is currently untreatable.

Abstract

A Northwestern researcher has collaborated with University of Alberta to develop a new gene therapy approach for treating corneal vascularization. They have tested an approach of overexpressing FOXC1 via adeno-associated virus (AAV) in mice. They found that the AAV-delivery of FOXC1, a protein with a fundamental role in vascular development and neovascularization, inhibits murine corneal vascularization in both pathological and physiological models, alkali burn injury and mice heterozygous for a mutation in Pax6, respectively. The inventors are preparing to transition to a clinical trial for this gene therapy, targeting patients with the corresponding Pax6 mutation. Additional patients suffering from corneal vascularization caused by infection, trauma or graft rejection, may also benefit from this therapy. Currently, the inventors are exploring the delivery of FOXC1 protein in eye drops using cell-penetrating peptides, liposomes and other analogous approaches.

Applications