An international team of scientists led by researchers at St. Jude Children’s Research Hospital has found a way to use CRISPR gene editing to help fix sickle cell disease and beta-thalassemia in blood cells isolated from patients. The study…provides proof-of-principle for a new approach to treat common blood disorders by genome editing.
“Our approach to gene editing is informed by the known benefits of hereditary persistence of fetal hemoglobin,” said Mitchell J. Weiss…”It has been known for some time that individuals with genetic mutations that persistently elevate fetal hemoglobin are resistant to the symptoms of sickle cell disease and beta-thalassemia…We have found a way to use CRISPR gene editing to produce similar benefits.”
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Recently, scientists have used several gene editing approaches to manipulate blood-forming stem cells for the possible treatment of sickle cell disease…All of these approaches remain untested in patients.
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At this stage, the scientists emphasize that it is still too early to begin clinical trials of the new gene editing approach…
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