Scientists use CRISPR to remove extra chromosome 21 from human cells

Scientists at Mie University in Japan have achieved a significant breakthrough in genetic research by using the CRISPR-Cas9 gene-editing system to remove an extra copy of chromosome 21 from human cell lines. This extra chromosome is the primary cause of Down syndrome, a condition occurring in approximately 1 in 700 live births.

Led by postdoctoral associate Ryotaro Hashizume in Dr. Shaonjong Park’s laboratory, the team developed specialized molecular guides that allow the CRISPR system to target only the additional copy of chromosome 21 while leaving healthy chromosomes intact. Following the removal, the cells exhibited nearly normal cellular functions, including improved survival rates, faster growth, and more efficient mitochondrial function.

The method proved successful in both stem cells and skin cells derived from patients with Down syndrome. While the research is currently limited to laboratory settings, the ability to remove an entire extra chromosome for the first time provides a potential foundation for future genetic therapies.