The latest discovery by Moran’s Mary Elizabeth Hartnett, MD, sheds light on potential new treatment approaches for age-related macular degeneration.
Mary Elizabeth Hartnett, MD, is at the forefront of efforts to find new treatments for age-related macular degeneration (AMD), a blinding disease affecting millions of people aged 55 and over each year.
Hartnett’s latest research study, published this month in Scientific Reports, identifies a significant new potential treatment path for the disease.
The study investigators provide evidence that delivering active Rap1a protein, using a cell-specific gene therapy approach, to a layer of cells in the eye known as the retinal pigment epithelium (RPE) protects against the wet (neovascular) form of AMD. It may also protect the RPE from stresses associated with increased risk of vision loss from AMD. Evidence suggests RPE dysfunction precedes the onset of both dry (atrophic) and wet AMD.
Using models, Hartnett tested two different RPE-specific promoters to increase active Rap1a in RPE and found one was superior to the other. Increased Rap1 reduced inflammation, cell death, and the formation of the abnormal blood vessels characteristic of neovascular AMD.
"Our study provides a potential future strategy where gene therapy could be used to increase active Rap1a in the RPE as a way to protect the retina from wet and potentially dry AMD," said Hartnett. "Additional studies are needed and are ongoing."
Additional authors on the study, "Optimal Inhibition of Choroidal Neovascularization by scAAV2 with VMD2 Promoter-driven Active Rap1a in the RPE," were: Haibo Wang, MD, PhD; Eric Kunz; Gregory J. Stoddard, PhD; and William W. Hauswirth, PhD.