Japanese Scientists Take Promising Step Toward Correcting Down Syndrome With Genetic Technique
- Dakila News
- Apr 17
- 2 min read
A visual summary summarizes the methodology. Credit: Editage Understand the news at your own pace: To make the content more accessible while maintaining technical depth, this news story was presented in two formats:
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Technical version: Aimed at readers with prior knowledge or professional interest in the topic.
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Accessible language:
For the first time, researchers in Japan have succeeded in removing the extra chromosome that causes Down Syndrome in human cells grown in the laboratory. Using a technique called CRISPR-Cas9, which works like “genetic scissors,” they eliminated the additional copy of chromosome 21, restoring normal cell function.
The research achieved a success rate of up to 37.5% in removing the extra chromosome, applying the technique to different types of cells, including those not actively dividing. This indicates that the approach can be practical in various situations.
Although the tests were only conducted in the laboratory, the results offer hope for the future. With further studies, this technique could lead to the development of treatments that correct Down's syndrome from the earliest stages of human development.
Technical language:
Japanese researchers from Mie University and Fujita Health University have achieved a significant milestone in genetics by using CRISPR-Cas9 technology to remove the extra copy of chromosome 21 in human cells grown in the laboratory. This trisomy is the main cause of Down Syndrome. The technique made it possible to restore normal gene expression in the treated cells, without affecting the original chromosomes inherited from the parents.
The study demonstrated up to 37.5% success in eliminating the additional chromosome, using pluripotent stem cells and skin fibroblasts. The approach proved effective in different cell types, including those not actively dividing, extending its therapeutic potential.
Despite the advances, the clinical application of this technique still faces challenges, such as ensuring the accuracy of the gene editing and avoiding unwanted side effects. However, the results open new prospects for developing genetic therapies that can correct trisomy 21 at early stages of human development.
A visual summary summarizes the methodology. Credit: Editage
Trisomic rescue via allele-specific multiple chromosome cleavage using CRISPR-Cas9 in trisomy 21 cells | PNAS Nexus | Oxford Academic
Study reveals genetic treatment for Down Syndrome
Genetic advances open up new possibilities for Down Syndrome - LabNetwork
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