CRISPR-based Gene Editing

Current treatment of exudative retinal diseases such as neovascular or “wet” age-related macular degeneration (AMD) and diabetic retinopathy includes intraocular injections of drugs that target vascular endothelial growth factor (VEGF). These anti-VEGF agents require frequent injections into the eye, which is costly and a burden to patients. Our laboratory demonstrated the use of novel CRISPR-Cas9 gene editing technology to permanently suppress VEGF secretion from human retinal cells. Using viral vectors that have been used safely in human gene therapy clinical trials, this technology has the potential to permanently suppress aberrant vessel growth (termed “angiogenesis”) in these retinal conditions.