Sla. Treatment at the DNA level could delay paralysis

A study conducted by the University of California has illustrated a method for restoring motor neuron function in people with Amyotrophic Lateral Sclerosis. Through a genetic editing operation and injecting antisense nucleotides into laboratory animals, the researchers have restored the expression of the STM2 protein, essential in motor neuron activity.

17 MAR – A team from the University of California has described, in Science, a method to restore the levels of the protein statmin-2 (STM2) in motor neurons, which is necessary to maintain the functionality of these nerve cells. This finding could delay the paralysis that occurs in people with Amyotrophic Lateral Sclerosis (ALS).

In all people with ALS and in nearly half of those with Alzheimer’s disease and frontotemporal dementia, a protein called TDP-43 loses its normal localization in the cell nucleus. This phenomenon leads to the loss of statmin-2, essential in the regeneration of neurons and in maintaining connections with muscle fibers, essential for movement.

Specifically, the researchers, through genetic editing, have isolated human gene sequences of STMN2 by injecting antisense nucleotides – which block the ability of RNA molecules to form a protein – into the cerebrospinal fluid in the animal model. The injection corrected statmin-2 pre-mRNA processing and restored protein expression, completely independent of TDP-43 protein functionality.

Source: Science 2023

March 17, 2023
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