RNA interference to target lipid disorders.

Abstract:

PURPOSE OF REVIEW:This review focuses on proof-of-principle experiments providing validation of new targets for the development of RNA interference-based therapeutics for dyslipidemia. RECENT FINDINGS:Over the past few years, RNA interference has become an accepted approach to manipulate gene expression in mammalian systems. Advantage has been taken of the relative tissue specificity of adenovirus for liver, and the genetic specificity of short hairpin RNA-mediated RNA interference to create liver-specific downregulation of different genes. A different approach to target liver has been through the administration of chemically modified short interfering RNAs. For example, apolipoprotein B messenger RNA has been silenced in liver and jejunum resulting in decreased plasma levels of apolipoprotein B and total cholesterol. SUMMARY:RNA interference has aroused great interest as a powerful experimental tool and a potential therapeutic strategy. Successful animal studies indicate that RNA interference might be useful for the treatment of various human diseases. Clinical studies will soon begin to assess the use of this new class of therapeutics to treat dyslipidemia.

journal_name

Curr Opin Lipidol

authors

Rondinone CM

doi

10.1097/MOL.0b013e3282ff861e

subject

Has Abstract

pub_date

2008-06-01 00:00:00

pages

285-8

issue

3

eissn

0957-9672

issn

1473-6535

pii

00041433-200806000-00011

journal_volume

19

pub_type

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