In a world-first, scientists at the University of California, Irvine have reprogrammed stem cells to fight, and potentially reverse brain diseases like Alzheimer's.
The edited cells were able to clear the junk without harming healthy tissue, leading to reduced inflammation and a dramatic improvement in brain performance in mice.
First, they grew human microglia
from stem cells, then used CRISPR gene editing (a technique that allows scientists to make precise changes to a cell's DNA) to reprogram the way they function.
Nearly 7 million Americans are living with Alzheimer's today, according to the Alzheimer's Association. Current treatments can only slow symptoms but not reverse them.
This latest breakthrough enabled the modified microglia, (an enzyme that breaks down brain plaque), from
accidentally harming critical, healthy parts of the brain. Plaques disrupt normal brain function by interfering with cell signaling, thus driving cognitive decline.
'Because the therapeutic protein was only produced in response to amyloid plaques, this approach was highly targeted yet broadly effective,' said lead-author Jean Paul Chadarevian, a postdoctoral researcher in the Blurton-Jones' lab.
'This work opens the door to a completely
new class of brain therapies,' said Robert Spitale, co-author and professor of pharmaceutical sciences.
'Instead of using synthetic drugs or viral vectors, we're enlisting the brain's immune cells as precision delivery vehicles.'
This new therapy could give hope to Alzheimer's patients and their families, but does have to go through multiple rounds of rigorous testing before it can be used to treat humans.
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