Abstract | BACKGROUND: Because a significant percentage of patients who require high-dose statin therapy for dyslipidemia experience treatment-related muscle symptoms and an inconsistent clinical response, alternative or adjunctive approaches to the management of dyslipidemia are needed. One alternative approach, antisense therapy, may offer an effective and well-tolerated option for patients not satisfactorily responsive to or intolerant to standard pharmacologic dyslipidemia therapies. OBJECTIVE: This review provides an overview of antisense technology and its potential role in the management of dyslipidemia. METHODS: Source material was obtained primarily from the published literature identified through a search of the PubMed database. RESULTS: Antisense technology is an evolving approach to therapy that has gone through a series of refinements to enhance molecular stability, potency, and tolerability. Mipomersen is an antisense molecule capable of producing clinically meaningful reductions in low-density lipoprotein cholesterol in patients with severe familial hypercholesterolemia. Further long-term clinical studies are required to more clearly quantify its impact on risk for cardiovascular events and establish whether it increases risk for hepatosteatosis. CONCLUSION: Antisense therapy represents a potentially effective and well-tolerated emerging treatment modality for numerous diseases. In the treatment of hypercholesterolemia, the antisense therapy mipomersen may provide a possible treatment option for patients with treatment-resistant dyslipidemia.
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Authors | Peter P Toth |
Journal | Journal of clinical lipidology
(J Clin Lipidol)
2011 Nov-Dec
Vol. 5
Issue 6
Pg. 441-9
ISSN: 1933-2874 [Print] United States |
PMID | 22108147
(Publication Type: Journal Article, Research Support, Non-U.S. Gov't, Review)
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Copyright | Copyright © 2011 National Lipid Association. Published by Elsevier Inc. All rights reserved. |
Chemical References |
- Apolipoproteins B
- Cholesterol, LDL
- Hypolipidemic Agents
- Oligonucleotides
- Oligonucleotides, Antisense
- mipomersen
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Topics |
- Apolipoproteins B
(blood)
- Cholesterol, LDL
(blood)
- Clinical Trials as Topic
- Drug Delivery Systems
- Drug Stability
- Dyslipidemias
(blood, therapy)
- Humans
- Hypolipidemic Agents
(therapeutic use)
- Oligonucleotides
(therapeutic use)
- Oligonucleotides, Antisense
(chemistry, therapeutic use)
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