Scientists in Japan have developed a method to treat Down syndrome. This was reported by the scientific journal PNAS Nexus here.
A research group at Mie University in the country developed a procedure that allows the removal of the extra chromosome 21, which causes Down syndrome.
In their work, the scientists used the CRISPR/Cas9 genome editing technology and adapted it to selectively remove only the "extra" copy of the chromosome.
Down syndrome occurs when human cells have three copies of chromosome 21 instead of the usual two. The additional genetic material leads to a wide range of disorders, from congenital heart defects to learning and speech difficulties.
The researchers developed an approach where CRISPR/Cas9 molecules target only the trisomic chromosome, leaving the others untouched. In editing lab-grown cells, they achieved complete elimination of the extra chromosome.
Subsequently, gene expression normalized: genes related to nervous system development became active, while the activity of genes responsible for metabolism decreased. Additionally, the cells began to grow and divide more rapidly.
