The authors of a new study from Mie University (Japan) say that the chromosome that causes Down syndrome can now be removed thanks to advanced gene editing technology.
Down syndrome affects approximately 1 in 700 newborns in the United States, caused by an extra copy of chromosome 21.
Children with Down syndrome often have multiple health problems such as congenital heart defects, thyroid disease, etc., mild to moderate intellectual disability, and difficulties in learning, language, and motor skills.
Researchers have long sought to address this chromosomal excess because current intervention methods do not address the root cause.
Down syndrome causes many health problems and also slows down the intellectual development of young children (Illustrative image: Pexels).
Recent work by Dr. Ryotaro's team from Mie University has shown a promising approach to CRISPR-based methods.
CRISPR-Cas9 is a flexible gene-editing system based on an enzyme that recognizes specific DNA strands. When the enzyme finds a suitable location, it cuts through the DNA strands. Scientists design CRISPR instructions to target only the unwanted chromosome.
This technique is called allele-specific editing, which helps direct the cutting enzyme to the correct location.
After the extra copy was removed, the edited cells showed improvements in gene expression. Genes involved in nervous system development became more active, and undesirable genes also decreased in activity.
Now, according to research published in the journal PNAS Nexus , CRISPR DNA editing technology can remove extra chromosomes in affected cells, bringing cellular behavior closer to normal function.
However, CRISPR can also affect healthy chromosomes. Researchers are refining the technology to target the extra copy of chromosome 21.
Dr. Hashizume, the lead researcher, hopes their work can be used to design regenerative therapies and treatments to address gene redundancy at its source.
Researchers will continue to analyze the risks of the DNA changes and monitor how the edited cells function over time, as well as their viability under real-world conditions.
Experts emphasize that this work highlights how CRISPR can help address genetic errors that cause conditions such as Down syndrome.
However, researchers still need to further assess the risks of large-scale DNA editing and ensure that the edited cells remain healthy under real-world conditions.
Although the technique is a new breakthrough, experts warn that more research is needed to ensure the safety and effectiveness of this method in different cell types and in living organisms.
Ethical questions surrounding gene editing in humans also need to be carefully considered before clinical application.
Source: https://dantri.com.vn/suc-khoe/dot-pha-khoa-hoc-da-co-the-loai-bo-nhiem-sac-the-gay-ra-hoi-chung-down-20250719144801016.htm
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