1 year later, first gene edited sickle cell patient is functionally cured

victoriagray
Victoria Gray. Credit: AFP

She’s the first person with a genetic disorder to get treated in the United States with the revolutionary gene-editing technique called CRISPR. 

And as the one-year anniversary of her landmark treatment approaches, [Victoria] Gray has just received good news: The billions of genetically modified cells doctors infused into her body clearly appear to be alleviating virtually all the complications of her disorder, sickle cell disease.

The researchers conducting the study Gray started caution that it’s too soon to reach any firm conclusions about the long-term safety and effectiveness of the approach… But Gray’s experience so far, along with two other patients who received the same treatment for a similar disorder, indicate the therapy has been effective for her and may work for other patients as well, they said.

Related article:  Brave new world? Why the public might be ready for gene-edited babies

Perhaps most importantly, the changes appear to have translated into significant health benefits for Gray. She hasn’t had any severe pain attacks since the treatment and hasn’t required any emergency room treatments, hospitalizations or blood transfusions.

In each of the previous two years, Gray had required an average of seven hospitalizations and emergency room visits due to severe pain episodes as well as requiring regular blood transfusions. She has also been able to reduce significantly her need for powerful narcotics to alleviate her pain.

“It’s a very big deal for me,” Gray said. “It’s a huge change.”

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