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Haemophilia : the Official Journal of the World Federation of Hemophilia|June 19, 2025
Evaluation of One-Stage Assays for the Monitoring of Recombinant Human Factor IX Padua Activity After Etranacogene Dezaparvovec Gene TherapyJan Astermark, Wolfgang Miesbach, Michiel Coppens, et al.
The Journal of Gene Medicine|May 12, 2009
AAV gene therapy as a means to increase apolipoprotein (Apo) A-I and high-density lipoprotein-cholesterol levels: correction of murine ApoA-I deficiencyStefan F C Vaessen, Robert Jan Veldman, Elisabeth M Comijn, et al.
Arteriosclerosis, Thrombosis, and Vascular Biology|July 9, 2005
Complete rescue of lipoprotein lipase-deficient mice by somatic gene transfer of the naturally occurring LPLS447X beneficial mutationColin J D Ross, Guoqing Liu, Jan Albert Kuivenhoven, et al.
The Journal of Biological Chemistry|March 18, 2003
Differential effects of scavenger receptor BI deficiency on lipid metabolism in cells of the arterial wall and in the liverMiranda Van Eck, Jaap Twisk, Menno Hoekstra, et al.
Human Gene Therapy|September 9, 2004
Long-term correction of murine lipoprotein lipase deficiency with AAV1-mediated gene transfer of the naturally occurring LPL(S447X) beneficial mutationColin J D Ross, Jaap Twisk, Janneke M Meulenberg, et al.
Human Gene Therapy|May 8, 2009
Effect of adeno-associated virus serotype and genomic structure on liver transduction and biodistribution in mice of both gendersAstrid Pañeda, Lucia Vanrell, Itsaso Mauleon, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|September 30, 2010
Sustained enzymatic correction by rAAV-mediated liver gene therapy protects against induced motor neuropathy in acute porphyria miceCarmen Unzu, Ana Sampedro, Itsaso Mauleón, et al.
Biochemical Pharmacology|September 2, 2003
P-selectin as a candidate target in atherosclerosisTom J M Molenaar, Jaap Twisk, Sonja A M de Haas, et al.
Proceedings of the National Academy of Sciences of the United States of America|April 25, 2002
Leukocyte ABCA1 controls susceptibility to atherosclerosis and macrophage recruitment into tissuesMiranda van Eck, I Sophie T Bos, Wolfgang E Kaminski, et al.
Human Gene Therapy|May 24, 2006
Correction of feline lipoprotein lipase deficiency with adeno-associated virus serotype 1-mediated gene transfer of the lipoprotein lipase S447X beneficial mutationColin J D Ross, Jaap Twisk, Andrew C Bakker, et al.
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