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Khabre desk
Recent reporting, analysis and explainers from the Gene Therapy desk.
health
A bioRxiv preprint describes a genetic switch that uses lenalidomide to trigger the death of engineered human T cells, including CAR T cells. The work reports preclinical cell experiments, not clinical testing.
health
A medRxiv preprint identifies a possible manufacturing mechanism behind REP/CAP DNA sequences found in the liver of a patient treated with the rAAV gene therapy Zolgensma. The proposed mechanism involves the placement of the AAV P5 promoter in a manufacturing plasmid.