By Nancy Lapid
(Reuters) -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.
HUMAN PATIENT RECEIVES FIRST 3D-PRINTED CORNEA
For the first time, a legally blind patient has received a 3D-printed cornea made of human eye cells in a successful start to an early-stage trial, implant manufacturer Precise Bio has reported.
Cornea transplants currently require eye tissue from human donors. As with all tissue and organ transplants, demand often outweighs the supply.
The 3D printing process allows hundreds of lab-grown implants to be derived from cells from a single donor cornea, the researchers said.
“For the first time in history, we’ve witnessed a cornea created in the lab, from living human cells, bring sight back to a human being,” lead surgeon Dr. Michael Mimouni of Rambam Medical Center in Israel said in a statement.
“It was… a glimpse into a future where no one will have to live in darkness because of a shortage of donor tissue.” Mimouni said.
The ongoing Phase 1 trial will ultimately test the PVEK corneal implant in 10 to 15 patients. Precise Bio, which is based in North Carolina and has operations in Israel, expects to announce initial results from the study in the second half of 2026.
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(Reporting by Nancy Lapid; Editing by Bill Berkrot)
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