Retinal degeneration treatments

For scientists pursuing retinal degeneration treatments, there is no shortage of culprits: More than 270 genes are known to cause the disease, which is a leading cause of blindness. That underscores the need to develop therapies that are broadly effective regardless of the gene defect. University of Florida Health researchers led by W. Clay Smith, Ph.D., an associate professor of ophthalmology research, have devised a modified protein to improve the function and survival of rod and cone photoreceptors.

The altered protein, arrestin1, works by boosting energy output in the photoreceptors. Improving their use of glucose slowed retinal degeneration and delayed vision loss in mouse models, the researchers found. The discovery may be broadly applicable to other inherited forms of retinal degeneration because the technique does not target the specific underlying genetic defect.

The findings were published Jan. 26 in the journal Human Gene Therapy.