Efficacy and long term safety of alipogene tiparvovec (AAV1-LPL(S447X)) gene therapy for lipoprotein lipase deficiency: an open label trial

We describe the 2-year follow-up of an open-label trial (CT-AMT-011-01) of AAV1-LPL(S447X) gene therapy for lipoprotein lipase deficiency (LPLD), an orphan disease associated with chylomicronemia, severe hypertriglyceridemia, metabolic complications and potentially life-threatening pancreatitis. The...

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Detalles Bibliográficos
Autores principales: Gaudet, Daniel, Méthot, Julie, Déry, Stéphane, Brisson, Diane, Essiembre, Christiane, Tremblay, Gérald, Tremblay, Karine, de Wal, Janneke, Twisk, Jaap, van den Bulk, Nick, Sier-Ferreira, Valerie, van Deventer, Sander
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2012
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4956470/
https://www.ncbi.nlm.nih.gov/pubmed/22717743
http://dx.doi.org/10.1038/gt.2012.43

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