Ophthalmology, Rare / Orphan
Gene Therapy Based Gene Targeting Approaches for the Treatment of Retinis Pigmentosa and Related Retinal Degenerations
2015 Gund Harrington Scholar
To many people, the words “genetic engineering” conjure up scary images of mutant humans, super-sized steer and weird half-fish, half-mouse creatures.
For scientists like Donald Zack, MD, PhD, Co-Director, Johns Hopkins Center for Stem Cells and Ocular Regenerative Medicine, genetic engineering means almost boundless opportunities for advancing the treatment of genetic diseases.
Dr. Zack, who also is the Guerrieri Professor of Genetic Engineering and Molecular Ophthalmology at Johns Hopkins, is profoundly interested in the application of genetic engineering to the treatment of retinal disease such as retinitis pigmentosa (RP) and glaucoma. He is an expert in the use of CRISPR, a powerful, revolutionary technology that makes manipulating human DNA more accessible and less costly.
Dr. Zack has been dedicated to ophthalmology and the biology of the eye since his days as a student at the Albert Einstein College of Medicine. “The dean was leaving to go to the Massachusetts Eye and Ear Infirmary, and he was trying to recruit young scientists to bring new ideas to the study of eye disease,” Dr. Zack remembers. “I knew I wanted to pursue a career in medical research, and he sold me on ophthalmology.”
That decision set his future course. Now, after more than 30 years spent studying the eye, Dr. Zack believes he and his research team may be on the threshold of a treatment breakthrough for retinitis pigmentosa and other forms of retinal disease. They are applying CRISPR technology to develop methods to actually modify the DNA within a patient’s eye.
“We are putting together the pieces to get enough of the picture to at least understand and develop a treatment,” Dr. Zack notes.
Although Dr. Zack has given up his clinical ophthalmology practice to focus on research, his firsthand experience with individuals with retinal disease and the sufferings they endure are imprinted in his memory bank.
“That’s what’s cool about this work; it combines basic science and the accumulation of knowledge with the clinical side,” he says. “If we can develop something to help patients, I would die happy.”