Gene Delivery Strategies for Corneal Disorders
Liujiang Song, PhD
Liujiang Song, PhD, is a scientist trained in both medicine and molecular therapy whose research lies at the intersection of adeno-associated virus (AAV) biology, gene delivery, and ocular therapeutics. AAV gene therapy has emerged as one of the most promising approaches for treating genetic diseases, owing to its ability to provide long-lasting expression and its proven success in multiple FDA-approved therapies. Dr. Song’s research aims to address critical unanswered AAV biology questions, lower effective vector doses and expand therapeutic applications. Her work involving engineering the core elements of AAV, with particular emphasis on the inverted terminal repeats (ITRs) — the only viral DNA sequences delivered to patients. In addition, she is extending AAV mechanisms to synthetic substrates, developing alternative delivery platforms capable of accommodating larger genes with full-length regulatory elements, thereby supporting physiological levels of gene expression and making gene therapy more broadly accessible.
A key effort in Dr. Song’s laboratory is advancing gene delivery strategies for anterior eye diseases, with a particular focus on corneal disorders. For example, in Fuchs endothelial corneal dystrophy — a genetic condition that affects the corneal endothelium and leads to progressive vision loss — her team is working to identify optimal AAV capsids, minimally invasive delivery routes, and targeted cell-specific transduction strategies to correct disease-causing mutations. By integrating innovations in AAV vector engineering with ocular applications, Dr. Song’s research seeks not only to halt disease progression but also to restore corneal health, offering a potential cure where corneal transplantation is currently the only option.