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Vol. 2, No. 3-4, 2005
Issue release date: January 2006
Section title: Therapeutic Strategies
Neurodegenerative Dis 2005;2:220–231
(DOI:10.1159/000089629)

Promises and Challenges in Developing RNAi as a Research Tool and Therapy for Neurodegenerative Diseases

Xia X.G. · Zhou H. · Xu Z.
Department of Biochemistry and Molecular Pharmacology, University of Massachusetts Medical School, Worcester, Mass., USA

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Article / Publication Details

First-Page Preview
Abstract of Therapeutic Strategies

Published online: 1/6/2006

Number of Print Pages: 12
Number of Figures: 5
Number of Tables: 1

ISSN: 1660-2854 (Print)
eISSN: 1660-2862 (Online)

For additional information: http://www.karger.com/NDD

Abstract

RNA interference (RNAi) is a recently discovered mechanism that is conserved in a wide range of eukaryotic species. Triggered by double-stranded RNA, RNAi identifies and destroys the mRNA that shares homology with the double-stranded RNA. Because of its specificity, RNAi has a high potential for being a powerful investigative and therapeutic tool. Indeed, its use as a reverse genetics tool to determine gene functions in invertebrates and cultured mammalian cells has already been experiencing an explosive growth. Gratifyingly we have also seen its application in dissecting neurodegeneration pathways in vitro. Although early studies suggested that RNAi could be readily adapted for in vivo studies in mammals using the transgenic technology, difficulties including low transgenicity and low RNAi efficacy have emerged, which has prevented the wide use of transgenic RNAi. The potential of RNAi therapy for human diseases has been a great source of excitement. Several new studies have demonstrated this concept in animal models of neurodegenerative disease. In this review, we highlight the recent literature and our own data in applying RNAi in research and therapy in the area of neurodegenerative diseases. We discuss the present and future challenges in the full realization of the potential for RNAi.


  

Author Contacts

Zuoshang Xu
Department of Biochemistry and Molecular Pharmacology
University of Massachusetts Medical School
364 Plantation St, 817 LRB, Worcester, MA 01605 (USA)
Tel. +1 508 856 3309, E-Mail zuoshang.xu@umassmed.edu

  

Article Information

Number of Print Pages : 12
Number of Figures : 5, Number of Tables : 1, Number of References : 73

  

Publication Details

Neurodegenerative Diseases

Vol. 2, No. 3-4, Year 2005 (Cover Date: Released January 2006)

Journal Editor: Nitsch, R.M. (Zürich)
ISSN: 1660–2854 (print), 1660–2862 (Online)

For additional information: http://www.karger.com/NDD


Article / Publication Details

First-Page Preview
Abstract of Therapeutic Strategies

Published online: 1/6/2006

Number of Print Pages: 12
Number of Figures: 5
Number of Tables: 1

ISSN: 1660-2854 (Print)
eISSN: 1660-2862 (Online)

For additional information: http://www.karger.com/NDD


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Copyright: All rights reserved. No part of this publication may be translated into other languages, reproduced or utilized in any form or by any means, electronic or mechanical, including photocopying, recording, microcopying, or by any information storage and retrieval system, without permission in writing from the publisher or, in the case of photocopying, direct payment of a specified fee to the Copyright Clearance Center.
Drug Dosage: The authors and the publisher have exerted every effort to ensure that drug selection and dosage set forth in this text are in accord with current recommendations and practice at the time of publication. However, in view of ongoing research, changes in goverment regulations, and the constant flow of information relating to drug therapy and drug reactions, the reader is urged to check the package insert for each drug for any changes in indications and dosage and for added warnings and precautions. This is particularly important when the recommended agent is a new and/or infrequently employed drug.
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