Abstract
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 lowdensity 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.
Keywords: LDL, HDL, Triglycerides, Familial hypercholesterolemia, Apolipoprotein AI, Lipoprotein lipase, (helperdependent) Adenovirus, Adeno-associated virus
Current Gene Therapy
Title: Gene Therapy in Disorders of Lipoprotein Metabolism
Volume: 7 Issue: 1
Author(s): Stefan F.C. Vaessen, Jaap Twisk, John J.P. Kastelein and Jan Albert Kuivenhoven
Affiliation:
Keywords: LDL, HDL, Triglycerides, Familial hypercholesterolemia, Apolipoprotein AI, Lipoprotein lipase, (helperdependent) Adenovirus, Adeno-associated virus
Abstract: 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 lowdensity 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.
Export Options
About this article
Cite this article as:
Vaessen F.C. Stefan, Twisk Jaap, Kastelein J.P. John and Kuivenhoven Jan Albert, Gene Therapy in Disorders of Lipoprotein Metabolism, Current Gene Therapy 2007; 7 (1) . https://dx.doi.org/10.2174/156652307779940261
DOI https://dx.doi.org/10.2174/156652307779940261 |
Print ISSN 1566-5232 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-5631 |
Call for Papers in Thematic Issues
Programmed Cell Death Genes in Oncology: Pioneering Therapeutic and Diagnostic Frontiers (BMS-CGT-2024-HT-45)
Programmed Cell Death (PCD) is recognized as a pivotal biological mechanism with far-reaching effects in the realm of cancer therapy. This complex process encompasses a variety of cell death modalities, including apoptosis, autophagic cell death, pyroptosis, and ferroptosis, each of which contributes to the intricate landscape of cancer development and ...read more
Related Journals
- Author Guidelines
- Graphical Abstracts
- Fabricating and Stating False Information
- Research Misconduct
- Post Publication Discussions and Corrections
- Publishing Ethics and Rectitude
- Increase Visibility of Your Article
- Archiving Policies
- Peer Review Workflow
- Order Your Article Before Print
- Promote Your Article
- Manuscript Transfer Facility
- Editorial Policies
- Allegations from Whistleblowers
- Announcements
Related Articles
-
Current HIV Treatment Guidelines - An Overview
Current Pharmaceutical Design Cross Talk Among Leukocytes, Platelets, and Endothelial Cells and its Relevance to Atherosclerosis and Coronary Heart Disease
Current Nutrition & Food Science A Possible Target of Antioxidative Therapy for Diabetic Vascular Complications-Vascular NAD(P)H Oxidase
Current Medicinal Chemistry Large Artery Stiffness and Antihypertensive Agents
Current Pharmaceutical Design Advanced Glycation End Products (AGEs), Oxidative Stress and Diabetic Retinopathy
Current Pharmaceutical Biotechnology Towards Prevention of Ovarian Cancer
Current Cancer Drug Targets Nuclear Magnetic Resonance Spectroscopy and Genetic Disorders
Current Medicinal Chemistry Phenomics of Vascular Disease: The Systematic Approach to the Combination Therapy
Current Vascular Pharmacology Drug-Induced Peripheral Neuropathy: Diagnosis and Management
Current Cancer Drug Targets Cross-Talk Between Mesenchymal Stem/Stromal Cells and Dendritic Cells
Current Stem Cell Research & Therapy Nutraceuticals - An Emerging Era in the Treatment and Prevention of Cardiovascular Diseases
Current Pharmaceutical Biotechnology Metabolic Syndrome the Gynecologist’s Approach
Current Women`s Health Reviews Design, Synthesis, and Biological Evaluation of Novel Tetramethylpyrazine- nitrone Derivatives as Antioxidants
Letters in Drug Design & Discovery Imaging Features of Thoracic Manifestations of Behçet’s Disease: Beyond Pulmonary Artery Involvement
Current Medical Imaging Heme Oxygenase-1 as a Target for the Design of Gene and Pharmaceutical Therapies for Autoimmune Diseases
Current Gene Therapy miRNAs Highlights in Stem and Cancer Cells
Mini-Reviews in Medicinal Chemistry Medicinal Properties of Mangiferin, Structural Features, Derivative Synthesis, Pharmacokinetics and Biological Activities
Mini-Reviews in Medicinal Chemistry Microbial Agents, Immune Function and Atheromatosis: The Chlamydophila pneumoniae Role
Current Immunology Reviews (Discontinued) The Estrogenic Burden on Vascular Risk in Male-to-Female Transsexuals
Current Pharmaceutical Design Insights into the Platelet Releasate
Current Pharmaceutical Design