CRISPR may hold key to curing muscular dystrophy

At 24, Benjamin Dupree has outlived many people with Duchenne muscular dystrophy…Doctors say the disease is terminal, but they tell you less about living with it…[I]n college, depression gripped him. “I didn’t know how I could keep going,” he says.

 

[T]he chance to end the pain and suffering of people like Dupree is CRISPR’s most compelling, if still distant, promise.  In early-stage lab experiments, academic scientists are showing that gene editing offers new ways to attack cancer, to knock out HIV and hepatitis infections, even to reverse blindness and deafness. Companies aren’t far behind. Three startups in the Boston area have already raised a combined $1 billion and partnered with some of the world’s biggest drug companies, like Bayer and Novartis.

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[In 2016, Eric Olson, biologist at the University of Texas Southwestern Medical Center,] showed he could repair mutations in mice with muscular dystrophy after sending viruses stuffed with CRISPR ingredients into their veins. “A mouse is not a boy, but we think we know exactly what needs to be done,” says Olson. If it works, he adds, “this is a cure, not a treatment.”

The GLP aggregated and excerpted this blog/article to reflect the diversity of news, opinion, and analysis. Read full, original post: Can CRISPR Save Ben Dupree?