Eye Program

By combining stem cell and gene therapy, microRNA, and complex genetics to uncover disease mechanisms and address pathology from various angles, our new therapies are advancing toward translation into clinical trials to treat common and complex eye diseases.

The Eye Program has:

• Developed the first successful gene therapy against delayed and abnormal wound healing in diabetic corneas using human organ cultures.
• Identified alterations of corneal epithelial stem cells in diabetes for the first time in a common eye disease and normalized these alterations with gene therapy.
• Recognized novel microRNAs as important functional regulators of normal and diabetic corneal wound healing.
• Designed a new system to produce corneal epithelial stem cells from iPSC that may have a significant impact in the treatment of stem cell deficiency-related blindness.
• Used genome-wide association studies (GWAS), identified a new promising gene candidate for keratoconus – lysyl oxidase – which has altered expression in diseased corneas.
• Successfully delayed retinal degeneration in two animal species using transplanted neural stem cells. This research is ready to be translated to clinical trials.

The Eye Program is directed by Alexander Ljubimov, PhD, FARVO. The program includes four other RMI principal investigators: Shaomei Wang, MD, PhD, Professor of Biomedical Sciences; Mehrnoosh Saghizadeh Ghiam, PhD, Associate Professor of Biomedical Sciences; Yaron Rabinowitz, MD, director of Division of Ophthalmology Research; and Kent Small, MD, research scientist. The team has secured five NIH grants, one CIRM grant and two industry grants. In addition, five other PhD researchers are associated with the program.

The Eye Program is collaborating with the Department of Neurosurgery to develop new polymeric nanodrugs for treating cancer and neovascularizing eye diseases. We are planning experiments, analyzing data and writing manuscripts.

The Eye Program is also collaborating with the Medical Genetics Institute on genome-wide association study (GWAS) analysis of keratoconus samples.