Gene therapy for lipid disorders

Current Controlled Trials in Cardiovascular Medicine

Volumn 1, Issue 2, 2000, Pages 120-127

  Kawashiri, M A ^a   Rader, D J ^a  

^a HOSPITAL OF THE UNIVERSITY OF PENNSYLVANIA   (United States)

Author keywords

Atherosclerosis; Cholesterol; Gene therapy; Lipoproteins; Vectors

Indexed keywords

ABC TRANSPORTER; ACID LIPASE; ALPHA TOCOPHEROL; APOLIPOPROTEIN A1; APOLIPOPROTEIN E; CHOLESTEROL; HIGH DENSITY LIPOPROTEIN CHOLESTEROL; LIPID; LIPID TRANSFER PROTEIN; LIPOPROTEIN LIPASE; LOW DENSITY LIPOPROTEIN RECEPTOR; PHOSPHATIDYLCHOLINE STEROL ACYLTRANSFERASE; TRIACYLGLYCEROL; VERY LOW DENSITY LIPOPROTEIN RECEPTOR;

ABETALIPOPROTEINEMIA; ADENO ASSOCIATED VIRUS; ANIMAL EXPERIMENT; ANIMAL MODEL; APHERESIS; ATHEROSCLEROSIS; CARDIOVASCULAR DISEASE; CAT; CHOLESTEROL ESTER STORAGE DISEASE; CLINICAL TRIAL; DISEASE ASSOCIATION; DYSLIPIDEMIA; EXPRESSION VECTOR; FAMILIAL HYPERCHOLESTEROLEMIA; GENE EXPRESSION; GENE MUTATION; GENE THERAPY; GENE TRANSFER; HUMAN; HYPERTRIGLYCERIDEMIA; MOLECULAR GENETICS; MOUSE; NONHUMAN; PATHOPHYSIOLOGY; RABBIT; REVIEW; SOMATIC CELL GENETICS; TANGIER DISEASE; VASCULAR DISEASE; WOLMAN DISEASE;

EID: 0034467613     PISSN: 14686708     EISSN: None     Source Type: Journal    
DOI: 10.1186/CVM-1-2-120     Document Type: Review

References (89)

1. Statin trials and goals of cholesterol-lowering therapy
2. Cholesterol management in theory and practice
3. Plasma triglyceride level is a risk factor for cardiovascular disease independent of high-density lipoprotein cholesterol level: A meta-analysis of population-based prospective studies
4. High-density lipoproteins - The clinical implications of recent studies
5. Familial hypoalphalipoproteinemia in premature coronary artery disease
6. Pathophysiology and management of low high-density lipoprotein cholesterol
7. Molecular genetics of the LDL receptor gene in familial hypercholesterolemia
8. Indications for low-density lipoprotein apheresis
9. Long-term improvement of hypercholesterolemia after ex vivo gene therapy in LDLR-deficient rabbits
10. In vivo correction of low density lipoprotein receptor deficiency in the Watanabe heritable hyperlipidemic rabbit with recombinant adenoviruses
11. Adenoviral delivery of low-density lipoprotein receptors to hyperlipidemic rabbits: Receptor expression modulates high-density lipoproteins
12. Hypercholesterolemia in low density lipoprotein receptor knock-out mice and its reversal by adenovirus-mediated gene delivery
13. Long-term reversal of hypercho-lesterolemia in low density lipoprotein receptor (LDLR)-deficient mice by adenovirus-mediated LDLR gene transfer combined with CD154 blockade
14. Effective treatment of familial hypercholesterolemia in the mouse model using adenovirus-mediated transfer of the VLDL receptor gene
15. Prolonged correction of hyperlipidemia in mice with familial hypercholesterolemia using an adeno-associated viral vector expressing very-low-density lipoprotein receptor
16. Construction and in vitro functional evaluation of a low-density lipoprotein receptor/transferrin fusion protein as a therapeutic tool for familial hypercholesterolemia
17. A pilot study of ex vivo gene therapy for homozygous familial hypercholesterolaemia
18. The familial hyperchylomicronemia syndrome
19. Severe hypertriglyceridemia, reduced high density lipoprotein, and neonatal death in lipoprotein lipase knockout mice
20. Induced mutant mice expressing lipoprotein lipase exclusively in muscle have subnormal triglycerides yet reduced high density lipoprotein cholesterol levels in plasma
21. A mutation in the lipoprotein lipase gene is the molecular basis of chylomicronemia in a colony of domestic cats
22. Lipoprotein lipase deficiency with pancreatitis in mink: Biochemical characterization and pathology
23. Phenotypic correction of feline lipoprotein lipase deficiency by adenoviral gene transfer
24. Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein
25. Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus
26. Long-term correction of obesity and diabetes in genetically obese mice by a single intramuscular injection of recombinant adeno-associated virus encoding mouse leptin
27. Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector
28. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector
29. Correction of hypertriglyc-eridemia and impaired fat tolerance in lipoprotein lipase deficient mice by adenovirus-mediated expression of human lipoprotein lipase
30. Adenovirus-mediated gene transfer of human lipoprotein lipase ameliorates the hyperlipidemias associated with apolipoprotein E and LDL receptor deficiencies in mice
31. Apolipoprotein E: Cholesterol transport protein with expanding role in cell biology
32. Familial apolipoprotein E deficiency
33. Mouse models of atherosclerosis
34. McClelland A. Phenotypic correction of hypercholesterolemia in apoE-deficient mice by adenovirus-mediated in vivo gene transfer
35. Apolipoprotein E deficiency in mice: Gene replacement and prevention of atherosclerosis using adenovirus vectors
36. Liver-directed gene transfer and prolonged expression of three major human apoE isoforms in apoE-deficient mice
37. Rapid regression of atherosclerosis induced by liver-directed gene transfer of apolipoprotein E in apoE-deficient mice
38. Complete atherosclerosis regression after human ApoE gene transfer in ApoE-deficient/nude mice
39. Prevention of atherosclerosis in apolipoprotein E-deficient mice by bone marrow transplantation
40. Pathogenesis of type III hyper-lipoproteinemia (dysbetalipoproteinemia): Questions, quandaries, and paradoxes
41. Familial combined hyperlipidemia workshop
42. Pathophysiology of hyperlipidemia in diabetes mellitus
43. The molecular pathology of lecithin: Cholesterol acyltransferase (LCAT) deficiency syndromes
44. Disruption of the murine lecithin: Cholesterol acyltransferase gene causes impairment of adrenal lipid delivery and up-regulation of scavenger receptor class B type I
45. ABC1: Connecting yellow tonsils, neuropathy, and very low HDL
46. The ABCs of cholesterol efflux
47. High density lipoprotein deficiency and foam cell accumulation in mice with targeted disruption of ATP-binding cassette transporter-1
48. Hypercatabolism of lipoprotein-free apolipoprotein A-I in HDL-deficient mutant chickens
49. Mutations in ABC1 in Tangier disease and familial high-density lipoprotein deficiency
50. Coronary heart disease prevalence and other clinical features in familial high-density lipoprotein deficiency (Tangier disease)
51. Genetic determinants of high density lipoprotein levels
52. Lack of apoA-I is not associated with increased susceptibility to atherosclerosis in mice
53. HDL deficiency in genetically engineered mice requires elevated LDL to accelerate atherogenesis
54. ApoA-I deficiency causes both hypertriglyceridemia and increased atherosclerosis in human apoB transgenic mice
55. Adenoviral vector-mediated overexpression of serum amyloid A in apoA-I-deficient mice
56. Tissue-specific expression of the human gene for lecithin:cholesterol acyltransferase in transgenic mice alters blood lipids, lipoproteins and lipases towards a less atherogenic profile
57. Expression of human lecithin:cholesterol acyltransferase in transgenic mice: Effects on cholesterol efflux, esterification, and transport
58. Overexpression of human lecithin cholesterol acyltransferase leads to hyperalphalipoproteinemia in transgenic mice
59. Lecithin:cholesterol acyltransferase overexpression generates hyperalpha-lipoproteinemia and a nonatherogenic lipoprotein pattern in transgenic rabbits
60. Correction of hypoal-phalipoproteinemia in LDL receptor-deficient rabbits by lecithin: Cholesterol acyltransferase
61. Potential gene therapy for lecithin-cholesterol acyltransferase (LCAT)-deficient and hypoalphalipoproteinemic patients with adenovirus-mediated transfer of human LCAT gene
62. High plasma HDL concentration associated with enhanced atherosclerosis in transgenic mice overexpressing lecithin-cholesteryl acyltransferase
63. Cholesteryl ester transfer protein corrects dysfunctional high density lipoproteins and reduces aortic atherosclerosis in lecithin cholesterol acyltransferase transgenic mice
64. Overexpression of lecithin: Cholesterol acyltransferase in transgenic rabbits prevents diet-induced atherosclerosis
65. Regression of atherosclerotic lesions by high density lipoprotein plasma fraction in the cholesterol-fed rabbit
66. Intravenous injection of rabbot apolipoprotein A-I inhibits the progression of atherosclerosis in cholesterol-fed rabbits
67. Inhibition of early atherogenesis in transgenic mice by human apolipoprotein AI
68. Human apolipoprotein A-I gene expression increases high density lipoprotein and suppresses atherosclerosis in the apolipoprotein E-deficient mouse
69. Apolipoprotein AI transgene corrects apolipoprotein E deficiency-induced atherosclerosis in mice
70. Inhibition of atherosclerosis development in cholesterol-fed human apolipoprotein A-I-transgenic rabbits
71. Somatic gene transfer of human apoA-I inhibits atherosclerosis progression in mouse models
72. Regression of atherosclerosis induced by liver-directed gene transfer of apolipoprotein A-I in mice
73. Gene therapy for atherosclerosis
74. Molecular defects underlying Wolman disease appear to be more heterogeneous than those resulting in cholesteryl ester storage disease
75. Genetic lipid storage disease with lysosomal acid lipase deficiency in rats
76. Targeted disruption of the mouse lysosomal acid lipase gene: Long-term survival with massive cholesteryl ester and triglyceride storage
77. Phenotypic correction of lipid storage and growth arrest in Woman disease fibroblasts by gene transfer of lysosomal acid lipase
78. Recent advances in elucidating the role of the microsomal triglyceride transfer protein in apolipoprotein B lipoprotein assembly
79. Knockout of the abetalipoproteinemia gene in mice: Reduced lipoprotein secretion in heterozygotes and embryonic lethality in homozygotes
80. Liver-specific inactivation of the abetalipoproteinemia gene completely abrogates very low density lipoprotein/low density lipoprotein production in a viable conditional knockout mouse
81. Hepatic overexpression of microsomal triglyceride transfer protein (MTP) results in increased in vivo secretion of VLDL triglycerides and apolipoprotein B
82. An adenoviral vector deleted for all viral coding sequences results in enhanced safety and extended expression of a leptin transgene
83. Humoral immunity to adeno-associated virus type 2 vectors following administration to murine and nonhuman primate muscle
84. Transient immunosuppression with FK506 permits long-term expression of therapeutic genes introduced into the liver using recombinant adenoviruses in the rat
85. Circumvention of anti-adenovirus neutralizing immunity by administration of an adenoviral vector of an alternate serotype
86. Prospects for the use of adeno-associated virus as a vector for human gene therapy
87. Adeno-associated virus as a vector for liver-directed gene therapy
88. Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors
89. Sustained expression of therapeutic level of factor IX in hemophilia B dogs by AAV-mediated gene therapy in liver