In a promising scientific breakthrough, Japanese researchers have succeeded in deleting an extra chromosome from human cells grown in the lab—opening new possibilities for developing future treatments for Down syndrome using modern gene-editing techniques.
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Down syndrome is caused by the presence of three copies of chromosome 21 instead of the usual two, leading to brain development disorders, learning difficulties, and various health issues.
A research team from Mie University in Japan used the CRISPR-Cas9 technique—known as the “molecular scissors”—to precisely remove the extra chromosome. The tool was able to distinguish the additional chromosome from the two inherited from the parents, allowing only the normal copies to be preserved.
After the correction process, the edited cells showed more balanced genetic behavior, especially in pathways related to brain development—indicating potential improvements in vital functions.
Despite the encouraging results, scientists emphasized that research is still in its early stages, and clinical application in humans will require many more years of study and experimentation.
Dr. Roger Reeves from the Johns Hopkins University School of Medicine explained that removing an extra chromosome from a single cell has been possible for over a decade, but CRISPR has significantly improved the precision of the process. However, he noted that the human body contains trillions of cells with the extra chromosome, making full-body treatment currently unrealistic.
The team tested the technique on two types of cells: induced pluripotent stem cells (reprogrammed from adult tissues) and skin fibroblast cells. They targeted multiple sites on the extra chromosome 21, forcing the cell to eliminate it. To increase the chances of success, they also suppressed the cells’ DNA repair mechanisms.
Still, only a small percentage of the millions of edited cells lost the extra chromosome. Experts believe the challenge remains immense, as effective treatment would require deleting the chromosome in hundreds of millions of cells—something not yet technically feasible.
The research still faces major technical and ethical hurdles, including concerns about unintended consequences and the potential misuse of the technology to create “designer babies.”
Nevertheless, researchers consider this breakthrough a vital step toward studying Down syndrome at the cellular level and paving the way for future therapeutic developments.
