Scientists have successfully tweaked the DNA in human heart cells to correct mutations that cause a deadly disease. If the gene-editing technique is proven safe, it could permanently cure children with a genetic disorder that leaves them wheelchair-bound by their early teens.
The genetic disease targeted in this study is called Duchenne muscular dystrophy (DMD), a disorder that affects about one in 5,000 males and causes the progressive wasting away of skeletal and heart muscles. Researchers took cells from patients with the disease, and used the gene-editing tool CRISPR-Cas9 to correct up to 60 percent of the mutations that cause the disease. That was enough to allow heart tissue engineered from those edited cells in a petri dish to...
from TechFishNews http://ift.tt/2DQfT8T
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