More than 100 thousand Americans, mostly African Americans, suffer from sickle cell disease, a painful, life-threatening disease that modern medicine still struggles to control.
Two gene therapies have been approved by the US Food and Drug Administration (FDA), including one that uses the Nobel Prize-winning technology CRISPR.
“These treatments for sickle cell disease represent a major advance in gene therapy,” said Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research.
“The potential for these products to change the lives of people with sickle cell disease is enormous,” he added.
CRISPR, a gene-editing technique, can change the DNA of animals, plants and microorganisms with extreme precision.
Its creators won the Nobel Prize in 2020.
The technology, which has breathtaking potential, has revolutionized the study of molecular life and has already led to experimental cancer treatments and drought-resistant crops.
Until now, the only cure for sickle cell disease, or sickle cell disease, was a bone marrow transplant.
Red blood cells normally move easily through blood vessels, but in sickle cell anemia they become crescent-shaped, or sickle-shaped, blocking blood flow and causing stroke, eye problems, infections, and severe pain.
A gene therapy using CRISPR was approved by the UK Medicines Regulatory Authority last month.
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2024-08-15 20:20:49
