Gene therapy and inherited dyslipidemia.

Endocrine Practice : Official Journal of the American College of Endocrinology and the American Association of Clinical Endocrinologists
T O'Brien

Abstract

To explore the current status and future potential of gene therapy for the inherited dyslipidemias. A brief overview of the inherited dyslipidemias, a review of the currently available means of transferring genetic material in vivo, and a discussion of two examples of conditions in which gene therapy may be useful--familial hypercholesterolemia and reduced high-density lipoprotein cholesterol syndromes--are presented. Although substantial progress has been made in the management of inherited dyslipidemia, optimal treatment regimens are not available in all cases. Gene therapy has recently emerged as a potential solution to some of these problems. For gene therapy to be successful, several factors are necessary: an efficient means of gene transfer, long-term transgene expression, and lack of toxicity. Although the feasibility of this approach has been demonstrated, currently available vectors have a number of technical and safety limitations. Gene therapy for inherited dyslipidemias has many technical hurdles that must be overcome before it will have widespread clinical application.

References

Mar 1, 1976·Metabolism: Clinical and Experimental·J D BrunzellE L Bierman
Apr 1, 1992·Proceedings of the National Academy of Sciences of the United States of America·B QuantinJ L Mandel
Apr 1, 1992·British Journal of Haematology·K Cornetta
Jun 11, 1992·Nature·A D Miller
Jun 11, 1992·Journal of Internal Medicine·S C Rall, R W Mahley
Aug 1, 1992·The Journal of Clinical Investigation·L D Stratford-PerricaudetP Briand
Aug 1, 1990·The Journal of Clinical Investigation·A R Tall
Nov 1, 1990·Proceedings of the National Academy of Sciences of the United States of America·J M WilsonJ R Chowdhury
Apr 1, 1990·The Journal of Clinical Investigation·E LeitersdorfH H Hobbs
Jan 1, 1989·Proceedings of the National Academy of Sciences of the United States of America·L F SoriaB J McCarthy
Apr 20, 1989·The New England Journal of Medicine·R H Eckel
Nov 9, 1989·The New England Journal of Medicine·D J Gordon, B M Rifkind
Oct 1, 1989·The American Journal of the Medical Sciences·K L BrighamL C Berry
May 20, 1988·Cell·C C ShihJ M Coffin
Oct 1, 1987·Proceedings of the National Academy of Sciences of the United States of America·T L InnerarityS M Grundy
May 1, 1986·Mayo Clinic Proceedings·B A KottkeS J Mao
Nov 1, 1985·Arteriosclerosis : an Official Journal of the American Heart Association, Inc·S YokoyamaA Yamamoto
Jun 1, 1972·The Journal of Clinical Investigation·T LangerR I Levy
Feb 1, 1980·European Journal of Clinical Investigation·A ChaitJ D Brunzell
May 15, 1993·Proceedings of the National Academy of Sciences of the United States of America·Y YangJ M Wilson
Jul 1, 1994·Nature Genetics·M S BrownD Steinberg
May 10, 1994·Proceedings of the National Academy of Sciences of the United States of America·Y YangJ M Wilson
Sep 30, 1993·Biochemical and Biophysical Research Communications·H HaddadaM Perricaudet
Dec 15, 1993·Proceedings of the National Academy of Sciences of the United States of America·A Kass-EislerL A Leinwand
Jan 1, 1994·The Medical Clinics of North America·E J Schaefer
Jul 1, 1993·The Journal of Clinical Investigation·L A KirshenbaumM D Schneider
Aug 1, 1993·The Journal of Clinical Investigation·B J RoesslerB L Davidson
Apr 1, 1993·Proceedings of the National Academy of Sciences of the United States of America·J Herz, R D Gerard

❮ Previous
Next ❯

Trending Feeds

COVID-19

Coronaviruses encompass a large family of viruses that cause the common cold as well as more serious diseases, such as the ongoing outbreak of coronavirus disease 2019 (COVID-19; formally known as 2019-nCoV). Coronaviruses can spread from animals to humans; symptoms include fever, cough, shortness of breath, and breathing difficulties; in more severe cases, infection can lead to death. This feed covers recent research on COVID-19.

Blastomycosis

Blastomycosis fungal infections spread through inhaling Blastomyces dermatitidis spores. Discover the latest research on blastomycosis fungal infections here.

Nuclear Pore Complex in ALS/FTD

Alterations in nucleocytoplasmic transport, controlled by the nuclear pore complex, may be involved in the pathomechanism underlying multiple neurodegenerative diseases including Amyotrophic Lateral Sclerosis and Frontotemporal Dementia. Here is the latest research on the nuclear pore complex in ALS and FTD.

Applications of Molecular Barcoding

The concept of molecular barcoding is that each original DNA or RNA molecule is attached to a unique sequence barcode. Sequence reads having different barcodes represent different original molecules, while sequence reads having the same barcode are results of PCR duplication from one original molecule. Discover the latest research on molecular barcoding here.

Chronic Fatigue Syndrome

Chronic fatigue syndrome is a disease characterized by unexplained disabling fatigue; the pathology of which is incompletely understood. Discover the latest research on chronic fatigue syndrome here.

Evolution of Pluripotency

Pluripotency refers to the ability of a cell to develop into three primary germ cell layers of the embryo. This feed focuses on the mechanisms that underlie the evolution of pluripotency. Here is the latest research.

Position Effect Variegation

Position Effect Variagation occurs when a gene is inactivated due to its positioning near heterochromatic regions within a chromosome. Discover the latest research on Position Effect Variagation here.

STING Receptor Agonists

Stimulator of IFN genes (STING) are a group of transmembrane proteins that are involved in the induction of type I interferon that is important in the innate immune response. The stimulation of STING has been an active area of research in the treatment of cancer and infectious diseases. Here is the latest research on STING receptor agonists.

Microbicide

Microbicides are products that can be applied to vaginal or rectal mucosal surfaces with the goal of preventing, or at least significantly reducing, the transmission of sexually transmitted infections. Here is the latest research on microbicides.

Related Papers

Journal of Thrombosis and Thrombolysis
J G PickeringJ M Isner
Nature Genetics
M S BrownD Steinberg
International Journal of Cardiology
D MehtaG D Angelini
© 2022 Meta ULC. All rights reserved