US approves sickle cell breakthrough with gene editing therapy
The United States on Friday approved a breakthrough medicine that uses revolutionary gene editing tool CRISPR to treat the debilitating blood disorder sickle cell disease.
More than 100,000 Americans, most of them Black, suffer from sickle cell disease, a painful, life-threatening illness that medical science has struggled to address.
US Food and Drug Administration (FDA) regulators approved two gene therapies, including one that uses the Nobel Prize-winning technology CRISPR.
“These treatments represent a major advancement in the field of gene therapy for patients with sickle cell disease,” Peter Marks, director of the FDA Center for Biologics Evaluation and Research, said.
“The potential these products have to transform the lives of patients living with sickle cell disease is enormous.”
CRISPR, a gene editing technique whose founders won a Nobel Prize in 2020, can change the DNA of animals, plants and microorganisms with extreme precision.
Hailed for its breathtaking potential, the technology has revolutionized the study of molecular life, already contributing to experimental cancer treatments and drought-resistant crops.
Until now, the only cure for sickle cell disease has been a bone marrow transplant.
Red blood cells normally move easily through blood vessels, but in sickle cell disease, they become crescent—or “sickle”-shaped—blocking blood flow and leading to strokes, eye problems, infections and severe pain.
Britain’s drugs regulator approved the gene therapy that uses CRISPR last month.
US approves sickle cell breakthrough with gene editing therapy (2023, December 8)
retrieved 8 December 2023
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