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Down syndrome (DS) is the commonest genetic cause of mental disability.
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Epigenetic modifications are implicated in neurocognitive dysfunction in DS.
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These epigenetic modifications are suitable therapeutic targets, but current therapies may have unwanted side effects.
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CRISPR-Cas9 is a precise genome editing technology that could be used to target epigenomic abnormalities in DS.
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A number of barriers − both technical and ethical − will need to be overcome for clinical translation.
- https://www.nature.com/news/should-you-edit-your-children-s-genes-1.19432
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