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Research

New approach could delay onset of blindness

04/06/2018
Scientists have discovered a new strategy that delayed the onset of blindness in mice, and also has the potential to prevent cell death in several neurodegenerative diseases.

The breakthrough was made while investigating inherited retinal disease, which has so far proven challenging for researchers due to the more than 4,000 different genetic mutations that can cause these conditions. However, many of these mutations have something in common – a propensity for creating misfolded proteins that cells in the eye can’t process.

As these proteins build up inside cells, they kill them from the inside out. But, by boosting the ability of cells to process misfolded proteins, they successfully were able to successfully prevent it from aggregating inside the cell.

“You can offer almost nothing in terms of treatment to a patient with retinitis pigmentosa or other inherited blindness today,” study senior author Professor Vadim Arshavsky said.

"The process successfully delayed the onset of blindness in mice"

“This investigation provides evidence that enhancing the capacity of the cell to process misfolded proteins is worth pursuing. Another important piece is that inherited blindness is just a subset of a larger category of neurodegenerative diseases, so this concept could be tested in other conditions, as well.”

Arshavsky’s team, in collaboration with colleagues from the California Institute of Technology, focused their research on the proteasome – the machinery found within a cell that eliminates misfolded proteins – within diseased mice.

The barrel-shaped structure of the proteasome functions like a paper shredder armed with hidden cutting elements.

Misfolded proteins pass through a “lid” on the shredder to be processed inside the cell, but diseased mice lack these entry points which enable the damaged proteins to build up.

However, instead of focusing on the shredders, Arshavsky and his team genetically increased the volume of these “lids”, which enabled the cells to process more misfolded proteins.

Arshavsky said that in humans, these “lids” could be added through drug compounds or gene therapy.

“If you can retain four times the number of the functional cells in the eye, that would mean decades more vision in a human patient,” Arshavsky said.

“It’s not a complete cure, but it’s a tremendous delay. This type of treatment has the potential to defer the onset of blindness beyond the human lifespan.”

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