Therapeutic Applications of Ribozymes

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Juni 1998



The purpose of Applications Therapeutic of Ribozymes is to provide an overview of the utility of ribozymes to selectively inhibit the expression of RNA. The ribozyme applications appearing in this book should benefit not only those experienced in ribozymes, but also those applying this ribozyme technology for the first time. It is hoped that a better understanding of the therapeutic application ofribozymes will have a significant impact on human disease in the near future. The field ofribozyme biochemistry has come a long way since the initial observation that RNA is capable of catalysis, initially in cis during processing of larger molecules and ultimately the use of ribozymes in trans to achieve cleavage of target sequence. The fundamental observation that hammerhead (and subsequently hairpin) ribozymes could be designed to cleave a target messenger RNA, and the clarification of sequence restrictions in both the ribozyme and the target RNA, have paved the ways for the use of ribozymes as a tool to manipulate gene expression at the molecular level. This had pre- ously been a domain of antisense oligonucleotides and triplex DNA. Sub- quent studies have explored the myriad biological systems in which ribozyme-mediated inhibition of genes involved in various cellular processes may be used to uncouple important signaling pathways or to reverse the p- notypic expression of a pathologic process.


Small Efficient Hammerhead Ribozymes
Philip Hendry, Trevor J. Lockett, and Maxine J. McCall

The Hairpin Ribozyme: Discovery and Development for Gene Therapy
Arnold Hampel

Molecular Manipulations of the Catalytic RNAs from the Human Hepatitis Delta Virus
N. Kyle Tanner

The Design, Production, and Validation of an Anti-HIV Type 1 Ribozyme
Lun-Quan Sun, Wayne Gerlach, and Geoff Symonds

Methods for Treating HIV by Gene Therapy Using Ribozymes
Eric M. Poeschla, Mang Yu, Mark C. Leavitt, and Flossie Wong-Staal

An Expression Vector for Multiple Ribozymes
Jun Ohkawa, Yutaka Takebe, and Kazunari Taira

Viral RNA Targets for Ribozymes: Influenza A Virus
Gerd Hobom and Annette Menke

Hammerhead Ribozyme-Mediated Cleavage of Hepatitis B Virus RNA
Fritz von Weizsäcker, Hubert E. Blum, and Jack R. Wands

Bovine Leukemia Virus Ribozymes
Glenn H. Cantor

Use of Ribozymes to Specifically Alter the MDR-1 Phenotype
David Y. Bouffard

Adenoviral-Mediated Anti-H-ras Ribozyme for the Treatment of Bladder Cancer
Akira Irie, Jerry Tsai, and Mohammed Kashani-Sabet

Tissue-Specific Delivery of an Anti-H-ras Ribozyme Against Malignant Melanoma
Tsukasa Ohkawa and Mohammed Kashani-Sabet

Ribozymes as a Novel Approach for the Treatment of Human Pancreatic Carcinoma
Hiroshi Kijima, David Y. Bouffard, and Kevin J. Scanlon

K-ras Ribozyme for Lung Cancer
Alex W. Tong, Yu-An Zhang, John Nemunaitis,
and Gabriele Mues

Anti-c-erb-B-2 Ribozyme for Breast Cancer
Toshiya Suzuki, Lisa D. Curcio, Jerry Tsai,
and Mohammed Kashani-Sabet

Ribozymes: Applications in Cancer Metastasis Research
Gunhild M. Mælandsmo, Inge Kjønniksen, Eivind Hovig, Kevin J. Scanlon, and Øystein Fodstad

Preformed Ribozymes: Strategies for Design and In Vivo Applicationas Anticytokine Agents
Mouldy Sioud

Folate-Polylysine-Mediated Delivery of a Multiunit Anti-BCR/ABL Ribozyme to BCR/ABL-Transformed 32D Cells
Lance H. Leopold, Scott K. Shore, Ramana M. V. Reddy, and E. Premkumar Reddy

Ribozymes Targeted Against AML1/MTG8: Evaluation of Ribozyme Efficiency in a Cell-Free System and Transfection with Cationic Liposome
Hiromichi Matsushita, Hiroyuki Kobayashi, Masahiro Kizaki, and Yasuo Ikeda

Ribozyme Targeting of the Growth Factor Pleiotrophin
Frank Czubayko, Anke M. Schulte, and Anton Wellstein

Ribozyme-Mediated Inhibition of Cell Proliferation: A Model for Identifying and Refining a Therapeutic Ribozyme
Thale C. Jarvis, Dennis Macejak, and Larry Couture

Inhibition of bAPP Synthesis by Ribozymes and Oligonucleotides in a Coupled Reticulocyte Transcription Translation System
Marilyn Smedman and Robert B. Denman

Selective Modification of Fatty Acid Synthesis Pathway by Expression of an Acetyl-CoA Carboxylase-Specific Ribozyme Gene
Joohun Ha

In Vivo Knockout of a Tissue-Specific Gene by Synthetic Ribozymes
S. Petter Lyngstadaas, Brian S. Sproat, Martina Blaschke, Janos Ludwig, Thomas Rupp, and Hans P. Prydz

Ribozyme-Mediated Downregulation of Gene Expression in Transgenic Mice
Shimon Efrat

Targeted Cleavage of RNA Using External Guide Sequences and Eukaryotic RNase P
Yan Yuan and Fenyong Liu

Application of In Vitro Selection in Design of External Guide Sequences That Direct Efficient Cleavage of RNA by RNase P
Yan Yuan

A PCR-Based Approach to Hammerhead Ribozyme Synthesis
Katalin Karikó and Elliot S. Barnathan

Measurement of Ribozyme Activity Using RNase Protection
P. Kamath and E. J. Shillitoe



". . . an impressive collection of ribozyme applications. . .a useful investment for anyone who contemplates or is actively using ribozymes for target RNA inactivations."-Nature Medicine
EAN: 9780896034778
ISBN: 0896034771
Untertitel: 'Methods in Molecular Medicine'. 1998. Auflage. Book. Sprache: Englisch.
Verlag: Humana Press
Erscheinungsdatum: Juni 1998
Seitenanzahl: 480 Seiten
Format: gebunden
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