Zobrazeno 1 - 2
of 2
pro vyhledávání: '"Paul P. A. Oranje"'
Autor:
Yvonne Mijnsbergen, Wim T. J. M. C. Hermens, Dennis Verbart, Pim Hermans, Jaap Twisk, Paul A. Dijkhuizen, Paul P. A. Oranje, Peter Verheesen, Janneke M. Meulenberg, Mark R. ten Haaft, Hans J. W. Haard
Publikováno v:
Molecular Therapy. 9:S162
Recombinant adeno-associated virus (rAAV) vectors hold promise for human gene therapy. Although most preclinical studies and current phase I clinical trials use vectors derived from rAAV serotype 2, other serotypes have been identified which transduc
Autor:
Michael R. Hayden, Janneke M. Meulenberg, Andrew C. Bakker, Maarten Beetz, Dennis Verbart, Jaap Twisk, John J.P. Kastelein, Fudan Miao, Colin J. D. Ross, Guoqing Liu, Karen van den Oever, Paul A. Dijkhuizen, Jan Albert Kuivenhoven, Paul P. A. Oranje, Wim T. J. M. C. Hermens
Publikováno v:
Molecular Therapy. 9:S17
Lipoprotein lipase (LPL) deficiency is characterized by excessive plasma triglyceride levels (TG), resulting in visibly milky plasma and severe clinical symptoms including life-threatening pancreatitis. There is no treatment for this inherited metabo