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United States: the first treatment using Crispr molecular scissors authorized

2023-12-08T18:33:11.573Z

Highlights: U.S. health authorities approve two treatments for sickle cell anemia. First treatment to use Crispr molecular scissors, a genome-editing technique. Crispr technology earned the Nobel Prize in Chemistry for France's Emmanuelle Charpentier and the American Jennifer Doudna in 2020. Sickle cell disease particularly affects African-Americans, the US agency points out.. Chronic anemia, acute pain attacks, and increased risk of infections are among the most common symptoms of sickle. cell disease.


These treatments will help fight sickle cell anemia, an inherited genetic blood disorder.


U.S. health authorities on Friday (Dec. 8) approved two treatments for sickle cell anemia, an inherited genetic blood disorder, including the first treatment to use Crispr molecular scissors, a genome-editing technique.

«

Gene therapy holds the promise of delivering more targeted and effective treatments, particularly for people with rare diseases," Nicole Verdun, head of the U.S. Food and Drug Administration (FDA), said in a statement.

"Transforming patients' lives"

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The potential of these products to transform the lives of patients living with sickle cell disease is enormous," he added. The development of these molecular scissors earned the Nobel Prize in Chemistry for France's Emmanuelle Charpentier and the American Jennifer Doudna in 2020.

Crispr technology has revolutionized genome manipulation with its accuracy and greater ease of use compared to previous tools. Both treatments, Casgevy and Lyfgenia, have been evaluated in clinical trials and are cleared by the FDA for patients as young as 12 years of age. The Casgevy treatment had already been approved in November by the British health authorities.

The patient's cells are modified using Crispr technology and then transplanted back into the individual. Sickle cell disease particularly affects African-Americans, the US agency points out. Chronic anemia, acute pain attacks, and increased risk of infections are among the most common symptoms of sickle cell disease. The repercussions, with damage to vital organs, can be serious or even fatal.

Source: lefigaro

All tech articles on 2023-12-08

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