American cured of anemia with CRISPR: it took only one injection

Two years ago, Olager was regularly hospitalized: his illness caused unpredictable

attacks of severe pain.Over the course of a year, that’s approximately 20 overnight hospitalizations and three visits to the intensive care unit. However, scientists managed to switch the genetic “switch” in Olager’s body to a new position - blood cells again learned to carry oxygen throughout the body.

“I did not expect that the changes would be so dramatic and almost instantaneous,” Olager comments on the result of therapy. “It really looks like a miracle.”

The technology that changed Olager's life, according to scientificcomparatively young. 10 years ago, a small group of scientists published a modest paper describing a simple but powerful invention for cutting specific DNA sequences. This became known as CRISPR gene editing.

Today, dozens of biotech companiesbuild their business on the potential of CRISPR. In the development of CRISPR therapy, the most advanced clinical trials, including the one Olager passed, Boston companies play a key role.

“Clinical developments reallyamazing,” said Feng Zhang, a scientist who invented CRISPR gene editing in his lab at the Broad Institute of Harvard and MIT.

Crispr Therapeutics and Vertex Pharmaceuticals,who made the same “infusion” that cured Olager, plan to submit their CRISPR therapy to the Food and Drug Administration later this year for registration.

Earlier this month, Crispr and Vertex announced thatall 31 people with sickle cell anemia who received treatment no longer experience severe attacks of pain. “I would be happy if I had a little less pain and fewer hospitalizations,” Olager said. But the result completely exceeded my expectations.”

Sickle cell anemia is caused by a mutationgene for hemoglobin, the protein that carries oxygen in the blood. To solve this problem, the scientists collected Olager's blood, used CRISPR to turn on the production of the so-called fetal hemoglobin that we all make in infancy, and then re-infused the blood cells back into his body.

In another trial testing the same therapyediting genes for a similar blood disorder called beta thalassemia, 42 of the 44 people who received treatment no longer needed the blood transfusion they needed to stay healthy. Two other people require far fewer blood transfusions than before.

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