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Article Search

Exagamglogene Autotemcel for Severe Sickle Cell Disease

Very exciting development in CRISPR-based therapy for transfusion-dependent sickle cell disease (SSD). 100% of patients avoided hospitalization for occlusive crisis. Long-term risks and concern for off-target gene editing will remain a concern. An argument can be made that for frequently admitted patients that this could be cost effective.

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