New breakthrough paves the way for gene therapy to treat amyotrophic lateral sclerosis

According to a VNA correspondent in Israel, a group of scientists at Tel Aviv University has just announced a breakthrough discovery that could open up an effective treatment direction for amyotrophic lateral sclerosis (ALS) - a rare motor neuron disease for which there is currently no cure.

The study, published this week in the journal Nature Neuroscience, uncovered a new molecular mechanism that causes ALS, and demonstrated the ability of RNA-based gene therapy to halt neurodegeneration and even regenerate damaged neurons.

ALS is a progressive degenerative disease that causes motor neurons to gradually die, leading to muscle weakness, paralysis, and eventually respiratory failure.

Current treatments only help slow disease progression and improve the patient's quality of life through medication, physical therapy and respiratory support.

The research was conducted in the laboratory of Professor Eran Perlson at Tel Aviv University, in collaboration with Sheba Medical Center and research institutes in Israel, France, Turkey and Italy.

The team focused on the protein TDP-43, which accumulates abnormally at nerve endings in ALS patients. The scientists found that healthy muscle cells secrete a small RNA – microRNA-126 – that prevents TDP-43 from forming toxic clumps.

In ALS patients, a sharp decrease in microRNA-126 causes TDP-43 to accumulate, leading to the destruction of mitochondria (the cell's energy source) and the gradual death of neurons.

Experiments also showed that adding microRNA-126 to patient and mouse tissues helped prevent neurodegeneration and promote motor nerve regeneration.

According to Professor Perlson, this discovery “opens up new treatment directions by restoring lost RNA signaling to protect nerve cells.”

The research team is currently developing a method to deliver microRNA-126 into the human body using AAV viral vectors, a technology that has been approved by the US Food and Drug Administration (FDA) in other gene therapies.

In addition to ALS, experts believe that this mechanism can also be applied to other neuromuscular diseases, or disorders related to toxic protein accumulation such as Alzheimer's.

Experts say the research results could also help detect ALS early before nerve damage becomes severe, and pave the way for the development of drugs or biological products that can activate or mimic the effects of microRNA-126.

Understanding how RNA signals from muscle control toxic protein accumulation could also help develop therapies for Alzheimer's and other protein-related disorders.

Professor Perlson affirmed that this discovery not only helps to understand how ALS starts but also opens up realistic hope for a gene therapy that can save patients' lives in the future./.

(TTXVN/Vietnam+)

Source: https://www.vietnamplus.vn/dot-pha-moi-mo-duong-cho-lieu-phap-gene-dieu-tri-benh-xo-cung-teo-co-mot-ben-post1076893.vnp