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Exa-Cel CRISPR Gene Therapy for Sickle Cell Disease Deemed Safe by FDA Advisory Panel

XTalks

CRISPR works as genetic scissors to edit parts of the genome. The scientists on the advisory committee were particularly focused on the CRISPR gene editing technology itself and how companies like Vertex that are developing CRISPR-based therapies are ensuring their treatments are not making off-target gene edits.