A young ALS patient playing computer games with the help of his electronic wheelchair

A man with amyotrophic lateral sclerosis, which affects neurons in the brain and spinal cord. A new method prevents formation of protein clumps often found in tissue samples taken from people who had the disease. Credit: Getty

Neurodegeneration

RNA snippets snare protein linked to degeneration of neurons

Technique ties up an innocuous protein before it gathers into dangerous clumps.

A disease-causing protein has been tricked into taking a molecular ‘bait’ that keeps the protein from clumping into poisonous little balls.

An otherwise useful protein found in most of the body’s tissues, TDP-43 sometimes malfunctions and forms neurotoxic clumps inside the brain. Such clumps are typically found in the brain tissue of people who have a degenerative brain condition, such as amyotrophic lateral sclerosis or Alzheimer’s disease.

Christopher Donnelly at the University of Pittsburgh in Pennsylvania and his colleagues designed a light-sensitive TDP-43 protein and introduced it into cultured human cells. When exposed to flashing light, the protein molecules gathered into clusters that were toxic to neuron-like cells.

TDP-43 usually binds to short strands of genetic material, so the researchers used short RNA molecules to snare the faulty proteins. These snared proteins were unable to damage cells.

The authors say that their approach could also help scientists to ‘bait’ proteins involved in other degenerative brain diseases, potentially leading to a new class of therapies.