ANTI-ANGIOGENIC GENE THERAPY FOR RETINAL DISEASE

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This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. This project was designed to test a potential genetic therapy for age-related macular degeneration (AMD), the most prevalent form of blindness in the elderly. In its most severe form AMD results in uncontrolled invasion of the retina by choroidal blood vessels leading to hemorrhage, retinal detachment and destruction of the macular retina causing central blindness. Efforts at therapy development have largely focused on stopping vessel growth with anti-angiogenic agents. The current state-of-the-art treatment involves monthly injection of anti-angiogenic antibodies into the vitreous. Although this treatment is effective in stopping disease progression in a majority of patients, frequent intraocular injections pose risk of infection and can present an unacceptable burden to some patients. We are testing a potential gene therapy that could require only one injection to maintain long-term therapeutic effectiveness. The use of nonhuman primates for these studies is critical because only monkeys and apes have a macula and other features closely resembling the human retina. It is our goal that these experiments will lead to an effective treatment for AMD in humans.