13/06/2026
Down syndrome -
Scientists removed the extra chromosome that causes Down syndrome from human cells using CRISPR gene-editing.
Led by Dr. Ryotaro Hashizume, the research team achieved "trisomy rescue" by targeting the surplus copy of chromosome 21, which is responsible for the condition's developmental and cognitive challenges.
By deploying precise molecular guides, the team successfully excised the extra chromosome and restored typical cellular functions in over 30% of the treated human cells. This breakthrough represents the first time an entire extra chromosome has been cleared from patient-derived cells, resulting in healthier, faster-growing cells with restored gene expression.
Although the research is in its early stages and currently confined to lab-grown cells, it represents a bold leap forward for precision medicine.
Instead of targeting individual genes, this approach addresses the genetic root of the condition, offering a revolutionary glimpse into future chromosomal therapies. Researchers caution that translating this laboratory success into real-world patient treatments will require extensive safety trials, refined delivery systems, and careful ethical considerations.
Nevertheless, this proof of concept marks a promising step toward potentially altering the trajectory of Down syndrome and other complex genetic conditions.
source: Hashizume, R., Wakita, S., Sawada, H., Takebayashi, S., Kitabatake, Y., Miyagawa, Y., Hirokawa, Y. S., Imai, H., & Kurahashi, H. Trisomic rescue via allele-specific multiple chromosome cleavage using CRISPR-Cas9 in trisomy 21 cells. PNAS Nexus.
Courtesy: Hashem Al-Ghaili
