By Ava Kikut
Scheie Vision Fall 2015
Artur Cideciyan, PhD, offers a cross-disciplinary approach to the study and treatment of hereditary retinal degenerative diseases. As a Research Professor of Ophthalmology at the Scheie Eye Institute’s Center for Hereditary Retinal Degenerations, he applies the rigor of mathematics and innovative engineering to the understanding of human diseases that cause genetic blindness. His work has led to major strides toward therapy for incurable vision loss.
Born in Istanbul, Turkey, Dr. Cideciyan moved to the United States at the age of 18 to attend the University of Miami. As an undergraduate, he became increasingly intrigued by the intersection of technology, science, and clinical practice. “I always loved the mixture of biology, physics, and medicine,” he explained.
After obtaining a bachelor’s degree in mechanical engineering, Dr. Cideciyan decided to pursue his master’s and doctorate degrees in biomedical engineering. As a PhD student, he took on a summer position at the Bascom Palmer Eye Institute in Miami. There, he met Dr. Samuel Jacobson, who has remained a close colleague ever since.
After working on a project involving the analysis of rhodopsin pigment in the human eye, Dr. Cideciyan developed an interest in retinal diseases. Inspired to contribute to the understanding and treatment of hereditary retinal degenerations, Dr. Cideciyan switched his dissertation focus from brain imaging to a little-understood feature in the retinas of female carriers of X-linked retinitis pigmentosa. He has remained in the retinal degeneration field for twenty-five years.
In 1995, Dr. Cideciyan moved his laboratory from Miami to Philadelphia, along with Dr. Jacobson, who came to establish the Center for Hereditary Retinal Degenerations at Scheie. Here, Dr. Cideciyan designs, develops, and evaluates outcomes of experimental treatments to blinding diseases, using advanced imaging, electrophysiology, psychophysics, mathematical modeling, and software development. He also works closely with scientists and clinicians at PennVet (specifically Drs. Gustavo Aguire and William Beltran). This collaborative team studies retinal diseases in animal models and evaluates therapeutic strategies on these models before they are tested on patients.
In 1997, Dr. Cideciyan began evaluating two distinct experimental therapies for a childhood blindness called Leber Congenital Amaurosis (LCA) after the discovery that the disease was caused by a mutation in the RPE65 gene. Along with Scheie colleagues, Dr. Cideciyan worked with two animal models affected by LCA, a genetically engineered mouse and a naturally occurring dog. This investigation found two therapies that could be successfully applied to both models: an oral vitamin-A like retinoid and gene therapy.
After almost a decade, both treatments progressed to human clinical trials. Within days of each type of treatment, patients showed large improvements in vision. “It was incredibly exciting and satisfying to be involved directly with testing different patients undergoing the different therapies,” Dr. Cideciyan remarked. During the trials, his lab studied vision changes before and after gene therapy. Results were published in high-level journals, including two publications in The New England Journal of Medicine and two in Proceedings of the National Academy of Sciences USA.
Over the past 15 years, Dr. Cideciyan has represented Scheie as the principal investigator in one of the first trials of an electronic chip implant to provide sight to patients with little or no vision. He has also intensively studied Stargardt disease, a common form of macular degeneration of young people caused by mutations in the ABCA4 gene. His research has led to several discoveries, including: the finding of a region of the retina in Stargardt patients that is naturally protected from the disease, the understanding of the consequences of supplementing patients with lutein, the invention of a new method for imaging melanin pigment in the eye, and the subsequent discovery that this pigment is counterintuitively elevated in eyes with Stargardt disease.
Dr. Cideciyan collaborated with researchers at Case Western Reserve University to produce the first animal model of Stargardt disease with a specific human mutation that causes severe symptoms to occur early in patients.
In addition to his reputation for innovative research, Dr. Cideciyan is admired by colleagues and patients for his compassion and amiability. “His gentle kindness toward patients with visual disability puts them at ease and always leads to their commenting about his humanity as well as his obvious intelligence,” remarked Dr. Jacobson. A member of the Scheie community for twenty years, Dr. Cideciyan continues to demonstrate an admirable sense of devotion to science and patient care.