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Science has managed to switch off the extra chromosome in Down syndrome. It has also opened the major ethical debate over gene editing

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2 min read
  1. Beth Israel Deaconess Medical Center researchers reported a modified CRISPR/Cas9 method that improved insertion of XIST into trisomy 21 cells.

  2. The approach silenced the extra chromosome 21 in a subset of tested cell lines, with XIST integration rates of about 20% to 40%.

  3. The work builds on earlier proof-of-concept studies and suggests a possible path toward treating Down syndrome at its genetic root.

  4. But the technique is still far from human use because of off-target effects, mosaicism, and incomplete editing, alongside major ethical concerns.

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