NIH R01 · 2024
Gyrate Atrophy Ocular and Systemic Study (GYROS)
1 Project Summary/Abstract for GYROS 2 Gyrate atrophy is a rare inherited chorioretinal degeneration that is associated with 3 hyperornithinemia caused by autosomal recessive mutations in the ornithine aminotransferase 4 (OAT) gene and leads to severe loss of vision. The current standard of care treatment is a 5 highly burdensome arginine-restricted diet (ARD) fraught with management complications. The 6 principal investigators are currently developing a gene augmentation therapy, a potential 7 treatment strategy that may preserve or improve vision while avoiding or reducing the 8 need for ARD. To facilitate a future interventional clinical trial, there is a need to characterize 9 the…
From the public funding record at NIH RePORTER. Describes the funded project, not the reviews below.