Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial

Gaudet, D, Méthot, J, Déry, S, Brisson, D, Essiembre, C, Tremblay, G, Tremblay, K, de Wal, J +4 more·2013·Gene Therapy
DOI 10.1038/gt.2012.43

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