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Parkinson’s gene editing breakthrough in mice offers hope for patients – their damaged neurons could be replaced

  • Scientists have discovered a way to convert mouse brain cells into neurons, replacing those damaged by disease
  • This could lead to a one-step strategy to treat a number of degenerative diseases that affect millions of people

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More than 10 million people worldwide are living with Parkinson’s. A new gene editing technique could create new neurons, replacing those lost to degenerative diseases such as Parkinson’s. Photo: Shutterstock
Agence France-Presse

Scientists have discovered a “one-step strategy” offering hope for treating Parkinson’s disease and other degenerative illnesses after converting mice brain cells into functioning neurons, they reported this week.

The motor defects associated with Parkinson’s are largely caused by the loss of dopamine-emitting neurons – electrically active cells that communicate with others – in the brain. A team of US- and China-based researchers used genome editing to convert a different type of brain cell – glial cells called astrocytes, for their star shape – into neurons, slowing the loss of dopamine and restoring motor functions in injured mice.

Past attempts to treat neurodegenerative disorders such as Parkinson’s have aimed to prevent or slow the loss of dopamine-emitting neurons, which do not naturally repopulate when they die or become damaged. The new technique simply aims to replace them through cell conversion, scientists said.
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“We’re now rebuilding the whole pathway,” Xiang-Dong Fu, a professor at the University of California, San Diego, said. “That becomes a very promising approach to turn non neuronal cells into neurons to replace those lost ones.”

Cellular and molecular biologist Xiang-Dong Fu is a professor at the University of California, San Diego. Photo: Fu Lab
Cellular and molecular biologist Xiang-Dong Fu is a professor at the University of California, San Diego. Photo: Fu Lab
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Worldwide, about seven per cent of people over 65 suffer from Alzheimer’s or some form of dementia, a percentage that rises to 40 per cent above the age of 85. More than 10 million people live with Parkinson’s.
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