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 beat again as a healthy heart would, according to a study published today in Science Advances.
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“I’m surprised by how simple and effective this method turned out to be so far,” [author Eric] Olson says. CRISPR seems to have cut only the DNA spots it was supposed to, without making any so-called off-target edits, he says.
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If studies continue to go smoothly, with promising results, clinical trials could start within a couple of years, according to Olson.
Read full, original post: New gene therapy could cure a deadly disease that stops the heart from beating
