Cover

Gene Therapy of Cochlear Deafness

Present Concepts and Future Aspects

Editor(s): Ryan A.F. (La Jolla, Calif.) 
Table of Contents
Vol. 66, No. , 2009
Section title: Paper
Ryan AF (ed): Gene Therapy of Cochlear Deafness. Adv Otorhinolaryngol. Basel, Karger, 2009, vol 66, pp 13-36
(DOI:10.1159/000218205)

Therapeutic Regulation of Gene Expression in the Inner Ear using RNA Interference

Maeda Y.a · Sheffield A.M.b · Smith R.J.H.a–c
aMolecular Otolaryngology Research Laboratories, Department of Otolaryngology, Head and Neck Surgery, bMedical Scientist Training Program, and cInterdepartmental PhD Genetics Program, University of Iowa, Iowa City, Iowa, USA

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Article / Publication Details

First-Page Preview
Abstract of Paper

Published online: 6/2/2009
Cover Date: 2009

Number of Print Pages: 24
Number of Figures: 0
Number of Tables: 0

ISBN: 978-3-8055-9035-8 (Print)
eISBN: 978-3-8055-9036-5 (Online)

Abstract

Targeting and downregulating specific genes with antisense and decoy oligonucleotides, ribozymes or RNA interference (RNAi) offer the theoretical potential of altering a disease phenotype. Here we review the molecular mechanism behind the in vivo application of RNAi-mediated gene silencing, focusing on its application to the inner ear. RNAi is a physiological phenomenon in which small, double-stranded RNA molecules (small interfering RNA, siRNA) reduce expression of homologous genes. Notable for its exquisite sequence specificity, it is ideally applied to diseases caused by a gain-of-function mechanism of action. Types of deafness in which gain-of-function mutations are observed include DFNA2 (KCNQ4), DFNA3 (GJB2) and DFNA5 (DFNA5). Several strategies can be used to deliver siRNA into the inner ear, including cationic liposomes, adeno-associated and lentiviral vectors, and adenoviral vectors. Transduction efficiency with cationic liposomes is low and the effect is transient; with adeno-associated and lentiviral vectors, long-term transfection is possible using a small hairpin RNA expression cassette.

© 2009 S. Karger AG, Basel


Article / Publication Details

First-Page Preview
Abstract of Paper

Published online: 6/2/2009
Cover Date: 2009

Number of Print Pages: 24
Number of Figures: 0
Number of Tables: 0

ISBN: 978-3-8055-9035-8 (Print)
eISBN: 978-3-8055-9036-5 (Online)


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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 government 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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