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Adeno-associated virus vector mediated gene therapy for Crigler Najjar syndrome: results of safety and efficacy from the dose escalation phase of the CareCN clinical trial

L. D'Antiga,U. Beuers,N. Brunetti-Pierri,U. Baumann,A. Di Giorgio, S. Aronson, A. Hubert, R. Romano,N. Junge,P. Bosma,G. Bortolussi,A. F. Muro, M. Pavlyuk,P. Veron,G. Ronzitti,F. Collaud, N. Knuchel-Legendre,F. Mingozzi,P. Labrune

HUMAN GENE THERAPY（2021）

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