Long-term correction of murine lipoprotein lipase deficiency with AAV1-mediated gene transfer of the naturally occurring LPLS447X beneficial mutation

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Veröffentlicht in:	Human gene therapy 2004-09, Vol.15 (9), p.906-919
Hauptverfasser:	ROSS, Colin J. D, TWISK, Jaap, HAYDEN, Michael R, MEULENBERG, Janneke M, GUOQING LIU, VAN DEN OEVER, Karin, MORAAL, Ewoud, HERMENS, Wim T, RIP, Jaap, KASTELEIN, John J. P, KUIVENHOVEN, Jan Albert
Format:	Artikel
Sprache:	eng
Schlagworte:	Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy Applied cell therapy and gene therapy Biological and medical sciences Biotechnology Errors of metabolism Fundamental and applied biological sciences. Psychology Gene therapy Health. Pharmaceutical industry Industrial applications and implications. Economical aspects Lipids (lysosomal enzyme disorders, storage diseases) Medical sciences Metabolic diseases Transfusions. Complications. Transfusion reactions. Cell and gene therapy
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