Gene-Edited Humans? Scientists Sound Alarm as UK Approves CRISPR Therapy to Treat Blood Disease
|Anonymous Coward (OP)
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11/20/2023 03:41 PM
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U.K. regulators this week approved the first CRISPR therapy to treat humans and U.S. regulators could approve the therapy — designed to treat blood disorders — as early as December. Meanwhile, the U.S. company behind another CRISPR technology, “base editing,” reported a successful initial study — despite 2 of 10 subjects experiencing heart attacks, resulting in one trial participant’s death.Quoting: Anonymous Coward 86340274
U.K. regulators on Thursday approved a therapy that uses CRISPR gene editing technology to treat two blood disorders. U.S. federal regulators are poised to approve that same treatment in December.
The exa-cel therapy, which goes by the brand name Casgevy, is the world’s first CRISPR therapy for humans to be approved for the market.
CRISPR is a gene editing technology that acts as a pair of “genetic scissors,” allowing scientists to edit sections of DNA by “snipping” specific portions of it and replacing them with new segments. First announced in a 2012 paper, CRISPR is celebrated as a cheap and easy way to edit genes.
Its inventors won the Nobel Prize in chemistry in 2020. In recent years, applications in plant manipulation and research on possible use in humans have proliferated as the technology has been promoted as a potential solution to problems running from disease, to food security to climate change.
But that research has been highly controversial, and a long series of papers has been published detailing the unintended effects of CRISPR gene editing, which has been found to produce many types of serious unintended DNA damage.
Casgevy is designed to treat two blood conditions: sickle cell disease and beta thalassemia. Sickle cell disease, also known as sickle cell anemia, most commonly occurs in people of African or Caribbean descent. It can cause debilitating pain.
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