UCL Team Develops Clinical-Scale Gene Editing for Wiskott-Aldrich Syndrome

A UCL-led team has created a clinical-scale CRISPR-Cas9-AAV6 workflow to correct Wiskott-Aldrich syndrome in hematopoietic stem cells, achieving a 61% gene correction rate. This development, which showed preserved cell function and limited genotoxicity in mice, represents a significant step towards potential gene therapy for the rare disorder. Long-term monitoring for rare alterations remains a consideration.