By Reda Wigle
Cutting-edge gene editing technology could eradicate Down syndrome, according to Japanese scientists.
Down syndrome, which causes a range of developmental differences and affects 1 in 700 newborns in the United States, is caused by the presence of an extra copy of chromosome 21.
The extra chromosome, also known as trisomy 21, causes cellular overactivity, compromises a range of processes within the body, and can manifest in distinctive physical traits, learning difficulties and health concerns.
Now new research out of Mie University in Japan suggests that by using the DNA-modifying tech CRISPR, it is possible to remove the surplus chromosome in affected cells and bring cellular behavior closer to typical function.
CRISPR-Cas9 is a gene-editing system that utilizes an enzyme to identify specific DNA sequences. Once the enzyme locates a matching site, it snips through the DNA strands.
Ryotaro Hashizume and his colleagues designed CRISPR guides to target only the trisomy 21 chromosome, a process called allele-specific editing, which directs the cutting enzyme to the desired spot.
When they used it on lab-grown cells, removing the extra copy of the gene normalized the way the genes expressed themselves in the body — suggesting that the genetic burden had been removed.
