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🚨 Scientists just took a huge step toward treating Down syndrome.
Japanese scientists have successfully used CRISPR-Cas9 gene editing to remove the extra chromosome responsible for Down syndrome in lab-grown human cells.
This process, known as trisomic rescue, targets and cuts the additional copy of chromosome 21—present in individuals with trisomy 21—encouraging the cell to eliminate the damaged chromosome entirely.
The result: cells that maintain a healthy pair of chromosomes, one from each parent, and exhibit normalized growth, gene expression, and reduced stress.
This marks a pivotal advance in the field of chromosomal disorder research. The technique worked not only in pluripotent stem cells but also in fibroblasts, suggesting wide applicability. While it’s not yet safe for clinical use due to potential off-target effects, the success of this method lays the groundwork for future treatments of Down syndrome and possibly other conditions caused by extra chromosomes, such as trisomy 13 and 18.
academic.oup.com/pnasnexus/arti…

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