Philadelphia, PA – Foundation Fighting Blindness has awarded Penn Vet Professor of Medical Genetics and Ophthalmology Gustavo D. Aguirre, VMD, PhD with a $230,000 grant to continue the Penn Translational and Research Facility.
“The main goal of the Translational and Research Facility is to accelerate the development and the pre-clinical testing of new and effective approaches to treat several forms of retinal degeneration (RD) in humans,” said Dr. Aguirre.
This initiative will continue to play a critical role in bridging the basic science research on RD conducted by investigators affiliated with the facility and the testing of new therapies in clinically relevant models. Special emphasis, said Dr. Aguirre, will be placed on developing gene therapy strategies that target photoreceptor cells; identifying molecular signals that favor either the death or survival of photoreceptors at the onset of disease; identifying new naturally occurringforms of RDs; and providing academic or industry investigators access to a technical platform and scientific counseling for testing potentially promising therapies.
“By examining the earliest cellular and molecular events that occur in the retina of the autosomal dominant and x-linked forms of retinitis pigmentosa models,” said Dr. Aguirre, “we expect to find new ways to stop the cascade of detrimental changes that take place before the photoreceptors are irreversibly committed to dying.”
Dr. Aguirre is a professor of medical genetics and ophthalmology at Penn Vet. He is a world-renowned researcher who, in 2001, led the research effort that restored vision to Lancelot, a dog who was born blind, through the use of gene therapy. Consequently, clinical trials in children were started for that type of blindness – Leber’s congenital amaurosis -- and so far have been showing efficacy and safety. After his success with treating Lancelot, Dr. Aguirre’s lab went on to study achromatopsia, and, with Dr. András Komaromy, assistant professor of ophthalmology at Penn Vet, serving as primary investigator, has also completed groundbreaking work in developing gene therapy to treat thattype of blindness. Dr. Aguirre earned his VMD and PhD degrees from the University of Pennsylvania. Together with Drs. Jacobson and Cideciyan of PENN's Scheie Eye Institute, and colleagues at the University of Florida's Powell Gene Therapy Center, Drs. Aguirre and Beltran are leading, respectively, gene therapy studies for the treatment of X-linked (RPGR) and autosomal dominant (Rhodopsin) retinal degeneration in preclinical models that very closely resemble the very severe and common human disorders.
About Foundation Fighting Blindness
The mission of the Foundation Fighting Blindness, Inc. is to drive research that will provide preventions, treatments and cures for people affect by retinitis pigmentosa(RP), macular degeneration, Usher syndrome and the entire spectrum of retinal degenerative diseases. Since its inception in 1971, the Foundation has raised more than $425 million to support these efforts. For more information, visit www.fightblindness.org.