CRISPR Gene Editing Enters Clinical Use Outside the US for the First Time, in Bahrain
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CRISPR gene editing, in which guide RNA directs the Cas9 enzyme to cut a target DNA sequence and correct disease-causing genes, has moved from laboratory to clinic. The FDA approved the first gene therapies for sickle cell disease in late 2023, targeting the defective gene behind that disorder. The technology has now been applied clinically for the first time outside the United States, in the Kingdom of Bahrain. The milestone matters for regions where sickle cell disease and thalassemia are common, since access to gene-editing treatments has so far been concentrated in the US. Open questions include how widely the approach can be deployed and how access will expand to other affected populations.
Written by a model from the reports · updated 2 hours ago
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- youtube.comCRISPR Gene Editing Reaches First Clinical Use Outside the US in Bahrain
A MedCram expert-analysis video examines CRISPR gene editing, in which guide RNA directs Cas9 to cut a target DNA sequence and correct disease-causing genes such as the defective gene in sickle cell disease. The FDA approved the first gene therapies for sickle cell disease in late 2023, and the technology has now been applied clinically for the first time outside the United States, in the Kingdom of Bahrain. The discussion covers how the mechanism works and why access matters in regions where sickle cell disease and thalassemia are common.
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