Gene Delivery for Corneal Repair

Liujiang Song, PhD

Dr. Song in lab Liujiang Song, PhD, is a scientist focused on developing gene therapy solutions for people affected by vision issues. Her research began with studying front-of-the-eye diseases, including corneal cloudiness, a major global cause of vision loss, that relies heavily on corneal transplants, which are limited by the availability of donor tissue and carry risks such as surgical complications and rejection. 

Why Gene Therapy? This approach offers a way to repair the eye at the genetic level. What makes gene therapy especially powerful is that it’s not restricted to an anatomic part of the eye. Whether the disease affects the cornea, retina, or optic nerve, the key is to identify the right genetic target and fix it, replace it, or introduce a healthy version in a safe and effective way.

A key effort in her lab is directed toward gene delivery, the process of transporting therapeutic genes into specific organs and cells. It’s often compared to package delivery: the gene is the package, and the vehicle, viral or non-viral, is the delivery truck. While the human body encounters many viruses, some harmful, some harmless, her team works with harmless kinds that naturally infect (enter) cells and deliver genetic material to specific parts of the eye, where they can restore functions or prevent further damage.

Delivery, however, is only step one. Another major part of her team’s work involves designing the genetic “package” itself, carefully assembling different molecular components (cis-elements) that work together, each with a specific role, to ensure the gene function where and when it’s needed. This level of control is key to making gene therapy safe, effective, predictable, and tunable.

Dr. Song will be actively collaborating with ophthalmologists on co-developing gene therapy solutions for eye diseases that cause vision loss. By combining clinical expertise with cutting-edge genetic tools, her team aims to accelerate the development of treatments that can preserve and restore vision.