A specific RNA molecule can halt and reverse the nerve cell damage of amyotrophic lateral sclerosis, a study in mice suggests, a discovery that may pave the way for effective future treatments for the fatal disease.
ALS, also known as Lou Gehrig's disease, affects nerve cells involved in movement and causes gradual paralysis of all muscles in the body.
The disease damages the neuromuscular junctions where nerve fibers meet muscle cells, but the mechanism underlying that damage has not been known until now, study leader Eran Perlson of Tel Aviv University said in a statement.
His team had previously observed that in ALS, a toxic protein called TDP-43 accumulates at nerve/muscle junctions and damages the mitochondria, the "energy factories" of nerve cells.
In healthy people, a molecule called microRNA-126 is produced by muscle cells and delivered to nerve cells to prevent TDP-43 from accumulating.
The researchers found that muscle production of microRNA-126 is reduced in ALS, they reported in Nature Neuroscience.
When they replenished microRNA-126 levels in ALS patients' muscle and nerve tissues in test tubes and in mice with the disease, toxic protein levels and nerve cell death were reduced and damaged nerves regenerated.
"Our findings may serve as a basis for developing an effective gene therapy focused on adding microRNA-126, which could bring hope to millions of patients and their families around the world," Perlson said.
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