|Articles|March 11, 2016

Two recent genetic discoveries may offer hope for patients with advanced AMD

The discovery of a gene for North Carolina macular dystrophy, PRDM13, and transplantation of induced pluripotent stem cells (iPSCs)-derived retinal cells show promise to restoring vision to patients with advanced AMD.

Take-home message: The discovery of a gene for North Carolina macular dystrophy, PRDM13, and transplantation of induced pluripotent stem cells (iPSCs)-derived retinal cells show promise to restoring vision to patients with advanced AMD.

Iowa City, IA-With age-related macular degeneration (AMD) as one of the major medical problems facing the developed world, this retina disease is expected to affect one in three persons over the age of 75 years in the United States by 2025.

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Therefore, treatment options must improve if clinicians are to address this disease adequately, according to Edwin Stone, MD, PhD.

Because more than 50% of AMD is genetic, the chances are good researchers eventually will understand the disease’s mechanism-thus allowing specific treatments to be developed and delivered to high-risk patients before irreversible disease develops.

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Recent inroads into AMD-such as the discovery of a gene for North Carolina macular dystrophy, PRDM13, and transplantation of induced pluripotent stem cells (iPSCs)-derived retinal cells-may restore vision to patients with advanced disease. Dr. Stone discussed these two important research developments.

Dr. Stone is professor of ophthalmology, Department of Ophthalmology and Visual Sciences, Stephen A. Wynn Institute for Vision Research, University of Iowa, Iowa City.

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