Approach could guide research for inherited blindness treatment in humans
The University of Florida, Department of Ophthalmology’s Dr. Ekaterina Lobanova is a leading author on study published in journal Nature Communications suggesting new approach to treat retinal degenerations in humans.
More than 2 million people worldwide live with inherited and untreatable retinal conditions, including retinitis pigmentosa, which slowly erodes vision.
Developing treatments is challenging for scientists, as these conditions are caused by more than 4,000 different gene mutations. But many of these mutations have something in common — a propensity for creating misfolded proteins that cells in the eye can’t process. These proteins build up inside cells eventually killing them.
Now scientists have shown that boosting the cells’ ability to process misfolded proteins could keep them from aggregating inside the cell. The researchers devised and tested the strategy in mice, significantly delaying the onset of blindness.
Their approach potentially could be used to prevent cell death in other neurodegenerative diseases, such as Huntington’s, Parkinson’s and Alzheimer’s.