A woman with sickle cell anemia has been cured of this genetic disease through a therapy based on the CRISPR gene editing tool, as explained in a speech at the III International Summit on Human Genome Editing, held in London between 6th and 8th of March.

The American patient Victoria Gray, had been suffering all her life from the complications of sickle cell anemia, the most frequent genetic disease in the world. This disease causes the red blood cells to have a different shape than normal: instead of being round they have a crescent shape. This causes them not to flow correctly through the blood vessels, where they get stuck, and cause severe pain throughout the body that, on many occasions, requires hospitalization, as well as various organic damages and a high risk of stroke.

In her speech, Gray, visibly moved, has told her story. In 2019, while in the hospital in one of the crises of the disease, she was offered an experimental treatment that consisted of obtaining stem cells from her bone marrow to modify them with gene editing and then reintroducing them into her body once corrected. She didn’t have to think about it too much, the answer was yes, she would participate in the clinical trial. Thus, the scientists extracted Gray’s cells to genetically modify them in the laboratory.

Once the cells were ready, Gray had to undergo a chemotherapy treatment to destroy her diseased cells, after which, in July 2019, the “super cells”, as she calls them, they were introduced into her body and regenerated her blood system.

“The life that I once felt like I was only existing in I am now driving in. I stand here before you today it’s proof that miracle still happen and that God and science can coexist.” “No longer have experienced severe pain and stopped my life just to be in the hospital for long periods of time.” For the first time she sees herself able to care for her children without help and look for a full-time job. Thus, Gray’s life has changed completely thanks to this revolutionary gene therapy.

However, not everything is as wonderful as it might seem in the wake of this case. The possibility that this therapy can be generalized to patients suffering from this disease seems remote today. The reason? Its price.

This type of treatment is among the most expensive in the world, with a cost of around 2.8 million euros, without counting the expenses derived from the necessary hospitalization and chemotherapy. This problem is compounded by the fact that more than 90% of sickle cell anemia cases occur in Africa and India.

Reducing the costs of this therapy is a challenge, but this goal must be part of the research in the development of these therapies.

 

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