TY - JOUR
T1 - Gene therapy in disorders of lipoprotein metabolism
AU - Vaessen, Stefan F. C.
AU - Twisk, Jaap
AU - Kastelein, John J. P.
AU - Kuivenhoven, Jan Albert
PY - 2007
Y1 - 2007
N2 - Current pharmacologic interventions in lipid metabolism are insufficient in a subset of patients at increased risk of cardiovascular disease. In particular, several monogenetic disorders of lipid metabolism with diverse clinical complications are beyond treatment to date. Somatic gene transfer is a potential approach to treat these disorders. This review describes the efforts made thus far to develop gene therapy for 3 major classes of dyslipidemia: Increased levels of low-density lipoprotein cholesterol, reduced levels of high-density lipoprotein cholesterol and increased plasma triglyceride levels. For many of the genetic causes underlying these conditions, proof-of-principle studies have been performed and in combination with improved vectors some of these strategies may be feasible for clinical use in the future
AB - Current pharmacologic interventions in lipid metabolism are insufficient in a subset of patients at increased risk of cardiovascular disease. In particular, several monogenetic disorders of lipid metabolism with diverse clinical complications are beyond treatment to date. Somatic gene transfer is a potential approach to treat these disorders. This review describes the efforts made thus far to develop gene therapy for 3 major classes of dyslipidemia: Increased levels of low-density lipoprotein cholesterol, reduced levels of high-density lipoprotein cholesterol and increased plasma triglyceride levels. For many of the genetic causes underlying these conditions, proof-of-principle studies have been performed and in combination with improved vectors some of these strategies may be feasible for clinical use in the future
U2 - https://doi.org/10.2174/156652307779940261
DO - https://doi.org/10.2174/156652307779940261
M3 - Review article
C2 - 17305527
SN - 1566-5232
VL - 7
SP - 35
EP - 47
JO - Current Gene Therapy
JF - Current Gene Therapy
IS - 1
ER -