Non-viral Gene Therapy Gene Design and Delivery

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K. Taira, K. Kataoka, T. Niidome (Eds.)

Non-viral Gene Therapy Gene Design and Delivery With 163 Figures, Including 25 in Color

Kazunari Taira, Ph.D Department of Chemistry and Biotechnology, Graduate School of Engineering The University of Tokyo 7-3-1 Hongo, Bunkyo-ku, Tokyo 113-8656, Japan and Gene Function Research Center National Institute of Advanced Industrial Science and Technology (AIST) Central 4, 1-1-1 Higashi, Tsukuba Science City 305-8562, Japan Kazunori Kataoka, Ph.D Department of Materials Science and Engineering, Graduate School of Engineering The University of Tokyo 7-3-1 Hongo, Bunkyo-ku, Tokyo 113-8656, Japan Takuro Niidome, Ph.D Department of Applied Chemistry, Faculty of Engineering Kyushu University 6-10-1 Hakozaki, Higashi-ku, Fukuoka 812-8581, Japan

Library of Congress Control Number: 2005925229 ISBN 4-431-25122-7 Springer-Verlag Tokyo Berlin Heidelberg New York This work is subject to copyright. All rights are reserved, whether the whole or part of the material is concerned, specifically the rights of translation, reprinting, reuse of illustrations, recitation, broadcasting, reproduction on microfilms or in other ways, and storage in data banks. The use of registered names, trademarks, etc. in this publication does not imply, even in the absence of a specific statement, that such names are exempt from the relevant protective laws and regulations and therefore free for general use. Product liability: The publisher can give no guarantee for information about drug dosage and application thereof contained in this book. In every individual case the respective user must check its accuracy by consulting other pharmaceutical literature. Springer is a part of Springer Science+Business Media springeronline.com © Springer-Verlag Tokyo 2005 Printed in Japan Typesetting: SNP Best-set Typesetter Ltd., Hong Kong Printing and binding: Shinano Inc., Japan Printed on acid-free paper

Foreword

Several years ago, when the discovery of catalytic RNA was recognized in a public manner, many people asked if new fields of therapy would soon be available. Although some tentative positive answers were given, nobody would say with certainty that RNA of various kinds was a truly promising means of altering gene expression. In fact, over the past decade, both our knowledge of RNAs with different functions and the utility of RNA in the inhibition or enhancement of gene expression have occurred with great drama. We proceeded in terms of possible therapeutic tools from RNase P and group I introns through “hammerhead” RNA enzymes, antisense technology, and more recently, to RNAi and its derivatives. A useful practical method of RNA delivery in animals will complete the picture. The diversity of RNA and the varied role of it inside cells and in therapy should be a tremendous challenge for young molecular biologists. This volume will make their task easier.

Sidney Altman Sterling Professor of Molecular, Cellular & Developmental Biology, Nobel Laureate Department of Molecular, Cellular and Developmental Biology Yale Univers