
Japanese breakthrough offers hope for treating D.o.w.n syndrome
(NLDO) - In a Japanese laboratory, scientists have successfully used CRISPR-Cas9 to correct errors in cell lines with Down syndrome for the first time.
A team led by geneticist Ryotaro Hashizume from Mie University (Japan) used the CRISPR-Cas9 gene editing system to successfully remove extra chromosomes from a triploid 21 cell line associated with Down syndrome.
Down syndrome is a genetic disorder caused by an abnormal number of chromosomes: an extra copy of chromosome 21, a condition called triploid 21.
In the experiment conducted in Japan, scientists used different types of cells with this syndrome, including pluripotent stem cells and skin fibroblasts.
The new technique has helped to precisely target the duplicate chromosome, ensuring that after removal, each cell retains one copy from each stem cell rather than two identical copies, according to the paper published in PNAS Nexus.
According to SciTech Daily, the breakthrough offers hope for a future treatment of Down syndrome, a condition that affects 1 in 700 children born.
Although the condition is easily diagnosed early in development, there is currently no treatment.
However, the authors also stressed that there is still a lot of work to do.
The method is not yet ready for use in living organisms, as it could also alter the remaining chromosomes, something they will have to study to find the right path.
However, the first, most difficult step has been conquered. The researchers believe that similar approaches could eventually be applied to neurons and glial cells.
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