Scientists claim to remove chromosome linked to Down syndrome
Japanese researchers from Mie University have developed a groundbreaking gene-editing approach that may eliminate the extra chromosome responsible for Down syndrome, according to findings published in PNAS Nexus.
Down syndrome, the most common chromosomal abnormality, is caused by an extra copy of chromosome 21 (trisomy 21). Using CRISPR/Cas9 technology, the scientists successfully removed the surplus chromosome from lab-grown cells, restoring normal gene expression. CRISPR, known for its DNA-cutting capabilities, was programmed to precisely target the extra chromosome through a technique called allele-specific editing, News.Az reports, citing foreign media.
Follow-up experiments revealed significant changes in gene activity: genes linked to nervous system development became more active, while those associated with metabolism slowed down. The treated cells also exhibited faster growth compared to untreated ones.
The team extended their testing to skin fibroblasts from individuals with Down syndrome, achieving similar results. However, researchers caution that the method is not yet ready for clinical use due to potential off-target effects on healthy cells. Work is ongoing to refine the process for greater precision and safety.
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