The Perelman School of Medicine at the University of Pennsylvania has launched the Penn Center for Advanced Retinal and Ocular Therapeutics (CAROT) to build on its previous success developing novel therapies for the personalized diagnosis and treatment of retinal and ocular disorders.
Worldwide, 285 million people suffer from visual impairment or blindness due to age-related macular degeneration, cataracts, open-angle glaucoma, diabetic retinopathy and more. Visual impairment is associated with a decreased quality of life and other problems such as autism spectrum disorders, cerebral palsy, hearing impairments and epilepsy. Still, relatively little funding is awarded to therapeutic approaches to restore vision among those patients.
Since 2007, Penn investigators, working closely with investigators at The Children's Hospital of Philadelphia (CHOP), have demonstrated groundbreaking success in dramatically improving vision using gene therapy for patients with a rare eye disorder known as Leber's congenital amaurosis (LCA) which renders children legally blind from birth. Through gene therapy, a new, healthy version of the gene causing LCA, RPE65, was delivered to the retina, ultimately leading to vision improvements in all 12 patients in the initial study.
Through its advanced research facilities and services, CAROT will extend these successful approaches developed for specific retinal or ocular disorders to support research on other disorders and advance therapies and cures for visual impairment and blindness through a focus on integration and partnership with others in the field of ophthalmology, neuroscience, cell biology, and genetics.
"A program like this unites all necessary approaches to researching and treating retinal and ocular disorders," says Jean Bennett, MD, PhD, director of CAROT and the F.M. Kirby Professor of Ophthalmology at the Perelman School of Medicine at the University of Pennsylvania. "An important premise of CAROT is to make the technologies that effectively advance research and therapies for the visually impaired and blind available to physicians, scientists, and patients."
The Center functions as a knowledge base to provide interested investigators with guidance on vector gene therapy construction, clinical trial design, patient identification, enrollment and regulatory oversight. It will also serve as the link between investigators and existing core services at the University of Pennsylvania, the NIH, CHOP, and other institutions that provide genotyping and bioinformatics, and other translational research services.
Beyond these research functions, CAROT engages biotech and pharmaceutical companies in ophthalmic genetic disorder research and therapeutic development, and will build a patient-focused advocacy program to guide clinical access and empower donors and foundations to contribute to advances in research.
Finally, the Center's leaders also aim to develop educational programs to ensure that the next generation of scientists, physicians and investigators are exposed to retinal and ocular disorders and access to new therapeutics.
A recipient of the 2011 NIH Director's Pioneer Award, Bennett has been recognized for her innovative approach and potential to produce an unusually high impact on this broad area of biomedical research.
"We believe the momentum we have established will allow us to develop an internationally recognized Center and become the premier center in the U.S. for the personalized diagnosis and treatment of retinal and ocular conditions," she says.