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Scientists have tried for years to lower the levels of the mutated protein behind the condition (red) without at the same time cleaning up their wild counterparts (blue), which are vital for brain health. Chinese researchers found four compounds (orange) that bind to the mutant protein and a crucial component of the internal mechanism of the neuron (light blue) for clearing invaders

Huntington's breakthrough as experts find a way to kill the mutated protein that causes irreversible brain damage

  • Chinese researchers found four compounds that can remove the mutated protein
  • The mutant huntingtin type is behind the paralyzing condition and kills neurons
  • The technique has only proven to work on human cells, mice and flies
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Hope has been awakened for thousands of Huntington's patients after scientists have found a possible way to treat the cruel disease.

Chinese researchers found four compounds that can remove the mutated protein behind the paralyzing state, which kills vital brain cells.

Their technique appeared to work only on human cells, mice and flies – but could open the door for the very first human treatment.

Scientists have tried for years to lower the levels of the mutated protein behind the condition (red) without at the same time cleaning up their wild counterparts (blue), which are vital for brain health. Chinese researchers found four compounds (orange) that bind to the mutant protein and a crucial component of the internal mechanism of the neuron (light blue) for clearing invaders

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Scientists have tried for years to lower the levels of the mutated protein behind the condition (red) without at the same time cleaning up their wild counterparts (blue), which are vital for brain health. Chinese researchers found four compounds (orange) that bind to the mutant protein and a crucial component of the internal mechanism of the neuron (light blue) for clearing invaders

Huntington harms the central nervous system and leads to involuntary movements, difficult talking and even memory loss.

On average, patients live between 10 and 20 years after their diagnosis. The NHS says there is currently no way to prevent it from getting worse.

The incurable condition, affecting 8,500 adults in the UK and around 30,000 in the US, occurs due to an error in the huntingtin gene.

This leads to an accumulation of the mutant huntingtin protein in neurons, causing them to die and cause devastating symptoms.

Scientists have been trying for years to lower the levels of the protein without at the same time cleaning up their wild counterparts, which are vital for brain health.

But until now, researchers had trouble making progress. The new findings are published in the prestigious journal Nature.

WHAT ARE HUNTINGTON & # 39; S?

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Huntington's disease is a disease caused by a defective gene in your DNA (the biological "instructions" that you experience that tell your cells what to do).

On average, patients live between 10 and 20 years after their diagnosis. The NHS says there is currently no way to prevent it from getting worse.

The incurable condition, affecting 8,500 adults in the UK and around 30,000 in the US, occurs due to an error in the huntingtin gene.

Huntington's can cause changes through movement, learning, thinking and emotions. Once the symptoms begin, the disease progresses gradually, so living with it means that you have to adapt to change one day at a time.

Huntington & # 39; s is not something you can catch; it is inherited. Every child naturally conceived by a parent carrying the Huntington gene has a 50 percent chance of inheriting it.

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Academics at Fudan University conducted various tests to find compounds that could remove the mutated protein without affecting normal ones.

Dr. Boxun Lu and the team found four promising ones, which reduced the number of defective huntingtins and had no influence on the others in all trials.

Tests on human neurons showed that fewer of them died when exposed to the molecules – dubbed 10O5, 8F20, AN1 and AN2.

Flies bred to have Huntington's and given the treatment were better able to climb and survived longer, compared to their untreated counterparts.

And mice with the rodent equivalent of the disorder also saw an advantage – they performed better on tests of their motor function than mice that were not treated.

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Experts who are not involved in the study, including Dr. Huda Zoghbi of the Baylor College of Medicine in Houston, welcomed the study.

She said that future trials should look for & # 39; off-target effects & # 39; including whether the compounds unintentionally clear up other vital proteins.

& # 39; Even effects that initially seem unimportant can build up in the course of long-term therapy and can become as problematic as the original toxic protein decades later, & # 39; she said.

But Dr. Zoghbi, who wrote in a linked editorial, added that the evidence & # 39; encouraging & # 39; was and said the treatment & # 39; functional improvements & # 39; yielded.

The compounds bind to the mutated protein and a crucial component of the internal mechanism of the neuron to clear up intruders.

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Professor John Hardy, a neuroscientist at University College London, said the study & # 39; interesting & # 39; but far removed from clinical studies.

Dr. Edward Wild, from the center of UCL in Huntington, said: “Unfortunately so far nothing that has worked in a mouse model has delayed the disease in patients.

& # 39; It is therefore important to study these new drugs thoroughly to get the best possible idea of ​​whether the approach is safe and effective enough to continue with human trials. & # 39;

Professor Tara Spiers-Jones, from the University of Edinburgh, said: & # 39; This is an interesting and potentially important article. & # 39;

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