Tokyo, July 25: Could gene editing hold the key to eliminating Down syndrome? Researchers at Japan’s Mie University in Tsu city have made a breakthrough using CRISPR-Cas9 technology to remove the extra chromosome responsible for the condition in lab-grown cells. By precisely targeting and deleting the surplus chromosome 21, they restored normal gene activity and improved cell function. While still in early stages, this discovery opens new possibilities for treating trisomy 21 at the genetic level.

As per a Sci Tech Daily report, Down syndrome is caused by the presence of an extra copy of chromosome 21, known as trisomy 21, which affects about 1 in every 700 live births globally. This additional genetic material disrupts normal development, leading to intellectual disabilities, distinct physical features, and increased risks for health issues such as heart defects, thyroid disorders, and early-onset Alzheimer’s disease. Currently, there is no cure or treatment that addresses the root genetic cause of the condition. What Is Artificial Blood? Know All About the Universally Compatible Lab-Made Blood Substitute Developed by Scientists in Japan.

The team at Mie University, led by Dr Ryotaro Hashizume, employed allele-specific CRISPR guides to precisely target only the extra chromosome 21, leaving the two normal copies intact. They tested the technique on pluripotent stem cells and skin fibroblasts derived from individuals with Down syndrome. Following gene editing, treated cells showed a significant shift in gene expression: genes involved in nervous system development were more active, while those linked to inflammation and metabolism were downregulated. Earthquake in Japan: Quake of Magnitude 5.5 on Richter Scale Jolts Tokara Islands, No Tsunami Warning Issued.

The edited cells also demonstrated faster growth and more typical behaviour compared to untreated cells. Despite these promising results, researchers caution that the approach is still experimental and not yet safe for clinical use, as reported by the New York Post. Potential off-target effects and the challenges of applying gene editing to complex tissues like the brain remain major hurdles.

(The above story first appeared on LatestLY on Jul 25, 2025 01:41 PM IST. For more news and updates on politics, world, sports, entertainment and lifestyle, log on to our website latestly.com).