Gene therapy could help in treating Down syndrome
Gene targeting could help in treatment for Down’s syndrome - interesting science news

Researchers have shown that targeting specific genes before birth might help in treatment of Down’s syndrome by reversing the brain maldevelopment and also improving the cognitive functioning.

The scientists developed two experimental models and used stem cells, which are cells that can turn into other cells in the brain. They found that inhibitory neurons were overproduced in these models of Down’s syndrome.

The gene targeted was the OLIG2 gene, which helped in rebalancing the excitatory-inhibitory balance towards a more healthy setting. Someday this approach could be used for treatment in humans.

Read the full story: Rutgers University
Scientific publication: Cell Stem Cell

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