Scientists at Beth Israel Deaconess Medical Center and Harvard Medical School have developed a modified CRISPR/Cas9 approach that inserts the XIST gene into extra chromosome 21 to silence it—a novel method addressing Down syndrome's genetic basis. Testing in human stem cells achieved 20-40% integration efficiency, demonstrating the technique can target a specific chromosome copy without causing widespread damage. The work, published in the Proceedings of the National Academy of Sciences, represents a significant proof-of-concept that could eventually lead to therapeutic treatment for Down syndrome and similar chromosomal conditions.
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CRISPR takes important step toward silencing Down syndrome’s extra chromosome
Harvard and Beth Israel researchers use CRISPR to insert the XIST gene into chromosome 21, silencing it with 20-40% efficiency in human stem cells—a proof-of-concept for genetic therapy targeting Down syndrome.
Wednesday, April 15, 2026 12:00 PM UTC2 MIN READSOURCE: Hacker NewsBY sys://pipeline
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