According to Japanese scientists, the editing technology of avant -garde genes could eradicate Down Syndrome.
Down Syndrome, which causes a variety of development differences and affects 1 in 700 newborns in the United States, is caused by the presence of an additional copy of chromosome 21.
The additional chromosome, also known as Trisomy 21, causes a cell overactivity, compromises a range of processes within the body, and can be manifested in distinctive physical features, learning difficulties and health problems.
Now, new research from the University of MIE in Japan suggests that, by using CRISPR DNA modification technology, it is possible to eliminate the surplus chromosome to the affected cells and to bring cellular behavior closer to the typical function.
CRISPR-CAS9 is a gene editing system that uses an enzyme to identify specific DNA sequences. Once the enzyme locates a place that matches, climbs through DNA chains.
Ryotaro Hashizume and his colleagues designed CRISPR guides to orient themselves in the Trisomy 21 chromosome, a process called the specific edition of the allele, which directs the cut enzyme to the desired place.
When they used it in laboratory grown cells, eliminating the additional copy of the gene normalized the way genes expressed themselves in the body, suggesting that the genetic load had been eliminated.
They also found that after removing the additional chromosome, genes related to the development of the nervous system were more active and those related to metabolism were less active. This is a backup of previous investigations that found additional copies of chromosome 21 disrupt the brain development during precocious fetal growth.
The researchers also tested their CRISPR guides in skin fibroblasts, which are mature cells and not the stem extracted from people with Down Syndrome.
In these fully developed cells, the editing method successfully eliminated the additional chromosome in several cases.
Following the removal, these corrected cells grew faster and had a shorter dubbing time than the un treated cells, suggesting that the elimination of the additional chromosome can help with the biological strain that slows the growth of cells.
But the CRISPR can also affect healthy chromosomes, and researchers are perfecting their program so that it is only attached to the additional copy of chromosome 21.
This work shows that instead of making small solutions, CRISPR can remove a whole chromosome.
Scientists published their findings in PNAS Nexus.
Hashizume and his team expect their work to be used to design regenerative therapies and treatments that treat genetic surplus at their source.
Researchers will continue to analyze the risks of DNA changes and oversee the operation of modified cells over time and their viability in the configuration of the real world.
A recent case study explored a medical mystery related to Down Syndrome; The brain of an American woman with Down Syndrome showed all the classic signs of Alzheimer’s disease, but remained free of symptoms throughout her life.
People with Down Syndrome have a much higher risk of developing a dementia related to Alzheimer’s as they grow older, about three to five times higher than the general population.
Scientists continue to work to identify the exact cause, but the additional copy of chromosome 21 is believed to lead to the overproduction of the amyloid precursor protein. This excess production leads to the accumulation of amyloid beta plates in the brain, a badge of Alzheimer’s disease.
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