Gene therapy for genetic lipid disorders: Lipoprotein lipase deficiency as a paradigm

Netherlands Journal of Medicine

Volumn 63, Issue 1, 2005, Pages 2-3

  Rader, D J ^a  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

LIPID; LIPOPROTEIN LIPASE; LOW DENSITY LIPOPROTEIN RECEPTOR; PARVOVIRUS VECTOR; TRIACYLGLYCEROL;

ACUTE PANCREATITIS; DRUG EFFICACY; EDITORIAL; EXPRESSION VECTOR; GENE EXPRESSION; GENE MUTATION; GENE REPLACEMENT THERAPY; GENE THERAPY; GENE TRANSFER; GENETIC DISORDER; HEMOPHILIA; HUMAN; HYPERLIPOPROTEINEMIA TYPE 1; HYPERTRIGLYCERIDEMIA; NONHUMAN; TREATMENT OUTCOME; TRIACYLGLYCEROL BLOOD LEVEL; VIRAL GENE DELIVERY SYSTEM;

GENE THERAPY; GENETIC VECTORS; HUMANS; HYPERLIPOPROTEINEMIA TYPE I; LIPOPROTEIN LIPASE; MUTATION;

EID: 13444274885     PISSN: 03002977     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Editorial

References (5)

1. Rader DJ, Tietge UJ. Gene therapy for dyslipidemia: clinical prospects. Curr Atheroscler Rep 1999;1:58-69.
2. Grossman M, Rader DJ, Muller DW, et al. A pilot study of ex vivo gene therapy for homozygous familial hypercholesterolaemia. Nat Med 1995;1:1148-54.
3. Nierman MC, Rip J, Twisk J, et al. Gene therapy for genetic lipoprotein lipase (LPL) deficiency: From promise to practice. Neth J Med 2004;63:14-9.
4. Lozier J. Gene therapy of the hemophilias. Semin Hematol 2004;41:287-96.
5. Manno CS, Chew AJ, Hutchison S, et al. AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood 2003;101:2963-72.