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

Autor: Gaudet, D1, Méthot, J1, Déry, S2, Brisson, D1, Essiembre, C2, Tremblay, G2, Tremblay, K1, de Wal, J3, Twisk, J3, van den Bulk, N3, Sier-Ferreira, V3, van Deventer, S3
Zdroj: Gene Therapy. Apr2013, Vol. 20 Issue 4, p361-369. 9p.
Databáze: Academic Search Ultimate