CRISPR Breakthrough:Scientists Successfully Remove Extra Chromosome Causing Down Syndrome
In a world-first scientific milestone, Japanese researchers have successfully used CRISPR gene-editing technology to remove the extra chromosome responsible for Down syndrome trisomy 21 from living human cells. Led by Dr. Ryotaro Hashizume at the National Cancer Center Research Institute in Tokyo, the team was able to eliminate the additional chromosome in more than 30% of treated cells. This correction restored the cells to a normal chromosomal state, offering new hope that the underlying genetic cause of Down syndrome could one day be targeted therapeutically.
The breakthrough was achieved using a precise CRISPR-Cas9 mechanism designed to cut and eliminate the entire extra chromosome without damaging the other two copies of chromosome 21. This is a dramatic leap forward, as previous efforts to treat or manage Down syndrome have focused only on symptoms never the source. While this development is still in its early stages and years away from clinical application, it lays the foundation for potential therapies that might correct the genetic imbalance at the root of the condition.
This success not only redefines what’s possible in gene therapy for Down syndrome, but also fuels broader conversations about future cures for other chromosomal disorders. It’s a stunning reminder of how far genetic science has come and how close we may be to rewriting the genetic errors once thought irreversible.
Source: National Cancer Center Japan, July 2025 | Research led by Dr. Ryotaro Hashizume
