Atherosclerosis: Gene Therapy


Knowledge of the human genome has opened up the possibility of developing gene therapy for several inherited and acquired diseases, such as atherosclerosis and its complications.

Keywords: gene therapy; atherosclerosis; inherited diseases; dyslipidemias; coronary heart disease


Grines CL, Watkins MW, Helmer G, et al. (2002) Angiogenic Gene Therapy (AGENT) Trial in patients with stable angina pectoris. Circulation 105: 1291–1297.

Grossman M, Rader DJ, Muller D, et al. (1995) A pilot study of ex vivo gene therapy for homozygous familial hypercholesterolemia. Gene Therapy 2: 587.

Isner JM, Vale PR, Symes JF and Losordo DW (2001) Assessment of risks associated with cardiovascular gene therapy in human subjects. Circulation Research 89: 389–400.

Laitinen M, Hartikainen J, Hiltunen MO, et al. (2000) Catheter‐mediated vascular endothelial growth factor gene transfer to human coronary arteries after angioplasty. Human Gene Therapy 11: 263–270.

Laitinen M, Mäkinen K, Manninen H, et al. (1998) Adenovirus‐mediated gene transfer to lower limb artery of patients with chronic critical leg ischemia. Human Gene Therapy 9: 1481–1486.

Laukkanen J, Lehtolainen P, Gough PJ, et al. (2000) Adenovirus‐mediated gene transfer of a secreted form of human macrophage scavenger receptor inhibits modified low density lipoprotein degradation and foam‐cell formation in macrophages. Circulation 101: 1091–1096.

Mäkinen K, Laitinen M, Manninen H, et al. (2002) Local adenovirus and plasmid–liposome‐mediated VEGF gene transfer to human lower limb artery after PTA; an interim analysis of a controlled, randomized trial. Molecular Therapy 5: 570–577.

Mann MJ, Whittemore AD, Donaldson MC, et al. (1999) Ex‐vivo gene therapy of human vascular bypass grafts with E2F decoy: the PREVENT single‐centre, randomised, controlled trial. The Lancet 354: 1493–1498.

Pakkanen TM, Laitinen M, Hippeläinen M, et al. (1999) Enhanced plasma cholesterol lowering effect of retrovirus‐mediated LDL receptor gene transfer to WHHL rabbit liver after improved surgical technique and stimulation of hepatocyte proliferation by combined partial liver resection and thymidine kinase ganciclovir treatment. Gene Therapy 6: 34–41.

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Ylä‐Herttuala S and Martin JF (2000) Cardiovascular gene therapy. Lancet 355: 213–222.

Further Reading

Friedmann T (1994) Gene Therapy. Fact and Fiction in Biology's New Approaches to Disease. New York, NY: Cold Spring Harbor Laboratory Press.

Gibbons GH and Dzau VJ (1996) Molecular therapies for vascular diseases. Science 272: 689–693.

Rekhter MD, Simari RD, Work CW, et al. (1998) Gene transfer into normal and atherosclerotic human blood vessels. Circulation Research 82: 1243–1252.

Rutanen J, Puhakka H and Ylä‐Herttuala S (2002) Post‐intervention vessel remodeling. Gene Therapy 9: 1487–1491.

Web Links

Apolipoprotein A‐I (APOA1); Locus ID: 335. LocusLink:

Lecithin‐cholesterol acyltransferase (LCAT); Locus ID: 3931. LocusLink:

Lipoprotein lipase (LPL); Locus ID: 4023. LocusLink:

Low density lipoprotein receptor (familial hypercholesterolemia) (LDLR); Locus ID: 3949. LocusLink:

Vascular endothelial growth factor (VEGF); Locus ID: 7422. LocusLink:

Very low density lipoprotein receptor (VLDLR); Locus ID: 7436. LocusLink:

Apolipoprotein A‐I (APOA1); MIM number: 107680. OMIM:‐post/Omim/dispmim?107680

Lecithin‐cholesterol acyltransferase (LCAT); MIM number: 606967. OMIM:‐post/Omim/dispmim?606967

Lipoprotein lipase (LPL); MIM number: 238600. OMIM:‐post/Omim/dispmim?238600

Low density lipoprotein receptor (familial hypercholesterolemia) (LDLR); MIM number: 606945. OMIM:‐post/Omim/dispmim?606945

Vascular endothelial growth factor (VEGF); MIM number: 192240. OMIM:‐post/Omim/dispmim?192240

Very low density lipoprotein receptor (VLDLR); MIM number: 192977. OMIM:‐post/Omim/dispmim?192977

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How to Cite close
Ylä‐Herttuala, Seppo(Sep 2006) Atherosclerosis: Gene Therapy. In: eLS. John Wiley & Sons Ltd, Chichester. [doi: 10.1002/9780470015902.a0006042]