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FDA approves the first CRISPR-based treatment

From From Bacterial Defence to Medicine: The CRISPR Gene-Editing Revolution

  1. Approval

    FDA approves the first CRISPR-based treatment

    Casgevy was approved for certain sickle-cell patients; cells are edited outside the body and returned after intensive conditioning.

    The FDA approved Casgevy for certain patients aged 12 and older with sickle-cell disease. Clinicians collect a patient’s blood-forming stem cells, edit them outside the body to increase fetal haemoglobin, and return them after intensive conditioning. The approval proved that CRISPR can deliver clinical benefit, but the process remains demanding, expensive and available only through specialised treatment centres.

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