Scientists from the University of Washington have been able to remove the extra chromosome 21 in cells taken from a person with Down Syndrome. In a gene therapy process that targets only the extra genetic material in the cell, scientist were able to successfully remove the chromosome 21 without damaging the integrity of the rest of the DNA in the nucleus.
Now I know some pro-lifers are immediately suspicious of this announcement thinking that it is meant to create a complete “cure” for Down Syndrome; a syndrome that many of us belief does not need a cure in the traditional sense. But looking closer, this technique was developed to help with some of the health problems of those with Down Syndrome, including an increased risk of leukemia. Dr. David Russell explains in an article on the breakthrough on RedOrbit:
“We are certainly not proposing that the method we describe would lead to a treatment for Down syndrome,” said study co-author Dr. David Russell, from the University of Washington’s Department of Medicine. “What we are looking at is the possibility that medical scientists could create cell therapies for some of the blood-forming disorders that accompany Down syndrome.”
These treatments could include treating Down syndrome patients with leukemia with genetically-modified stem cells that are derived from their own cells, but without the extra chromosome. Stem cells could be taken from the bone marrow of the patients, doctors could remove the extra chromosome, and then the healthy cells could then be grown and transplanted back into the patient.
Russell added that his team’s findings could not only lead to new treatments for symptoms of the disease, they could also help geneticists to better understand the underlying causes of the disease and any potential treatments or preventions they might be able to pursue.
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This is really good news not only for those with Down Syndrome and for all those who live with any kind of trisomy and for the field of gene therapy in general. One of the dangers of gene therapy has always been that the therapy will damage the DNA in the cell in a way that will cause side effects like cancer. The fact that the researchers where able to remove the chromosome without damaging, breaking or rearranging other chromosomes in the nucleus is a major achievement.