new Hope! Two studies reveal the pathogenesis and new therapies of "cold fever"

Release date: 2019-12-27 Views: 0

Source: Biological Exploration  

Amyotrophic lateral sclerosis (ALS, also known as motor neuron disease, gradual freezing disease), along with cancer and AIDS, has been listed by the World Health Organization as "the world's top five incurable diseases", and there is currently no possibility of cure. According to statistics, by the end of 2010, there were already about 200,000 ALS patients in China, and the number of patients will increase by 18,000 each year, and about 80% of them will die within 3 to 5 years after the onset of illness.

Although the etiology and pathology of amyotrophic lateral sclerosis is not clear, two reports recently published in international leading journals have once again strengthened people's confidence in fighting ALS and brought new hope to patients.

New pathogenesis of ALS may be discovered

Scientists have been trying to find the cause of amyotrophic lateral sclerosis from various aspects, so as to prescribe the right medicine. On December 18, a new study published in Science Translational Medicine gave people a better understanding of the pathogenesis of ALS.

The report states that microRNAs may play a role in the pathophysiology of ALS, miR-218 controls neuronal activity by regulating potassium channel Kv10.1, and miR-218 expression is reduced in motor neurons in ALS patients.

During the experiment, researchers found that miR-218 is high in healthy human motor neurons, but it is down-regulated in ALS motor neurons, and its mRNA targets are up-regulated (inhibited) accordingly. The researchers then screened thousands of ALS genomes and identified six rare variants in the human miR-218-2 sequence, and found that miR-218 gene variants failed to regulate neuronal activity, meaning that this small Endogenous RNA is important for neuron robustness.

The researchers said that miR-218 activity in motor neurons may be susceptible to human ALS failure, suggesting that miR-218 may be a potential therapeutic target for amyotrophic lateral sclerosis.

The most effective ALS therapy to date

If the report published in the "Science" sub-journal points the way for scientists to explore ALS treatment in the future, a research report published in "Nature Medicine" 5 days later clearly proposed a "currently most effective" ALS therapy: Inject a virally-depressed gene silencing agent to prevent ALS from degeneration, thereby suppressing the disease for a long time.

Studies have shown that mutations in the SOD1 gene can lead to inefficient removal of superoxide radicals or create other toxicity that can cause motor neuron cell death, leading to ALS. In this new study, researchers injected a single injection of a virus carrying shRNA, an artificial RNA molecule capable of silencing or shutting down a target gene, into two sites in the spinal cord of undeveloped adult mice that express the SOD1 gene causing mutations in ALS. On the point, it was found that the neurodegeneration of the mice became significantly relieved before the symptoms and showed normal neural function. In addition, in mice that have developed symptoms of ALS, the injection effectively prevented the further development of the disease and the degradation of motor neurons.

In subsequent experiments, researchers found that using an injection device developed for adults can reliably perform surgery without surgical complications.

Scientists have never stopped on the way to unlocking the mysteries of disease and its treatment. As more and more researchers invest in ALS, it is believed that there will be more alternative therapies to help patients fight the disease.


[1] Injectionof virus-delivered gene silencer blocks ALS degeneration, saves motor function

[2] Humangenetics and neuropathology suggest a link between miR-218 and amyotrophiclateral sclerosis pathophysiology

[3] Spinalsubpial delivery of AAV9 enables widespread gene silencing and blocksmotoneuron degeneration in ALS

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