Therapeutic Applications of Ribozymes

1. Front Matter

   Pages i-xiv

   PDF

2. Small Efficient Hammerhead Ribozymes

   • Philip Hendry, Trevor J. Lockett, Maxine J. McCall

   Pages 1-15

3. The Hairpin Ribozyme

   • Arnold Hampel

   Pages 17-28

4. Molecular Manipulations of the Catalytic RNAs from the Human Hepatitis Delta Virus

   • N. Kyle Tanner

   Pages 29-49

5. The Design, Production, and Validation of an Anti-HIV Type 1 Ribozyme

   • Lun-Quan Sun, Wayne Gerlach, Geoff Symonds

   Pages 51-64

6. Methods for Treating HIV by Gene Therapy Using Ribozymes

   • Eric M. Poeschla, Mang Yu, Mark C. Leavitt, Flossie Wong-Staal

   Pages 65-82

7. An Expression Vector for Multiple Ribozymes

   • Jun Ohkawa, Yutaka Takebe, Kazunari Taira

   Pages 83-95

8. Viral RNA Targets for Ribozymes

   • Gerd Hobom, Annette Menke

   Pages 97-110

9. Hammerhead Ribozyme-Mediated Cleavage of Hepatitis B Virus RNA

   • Fritz von Weizsäcker, Hubert E. Blum, Jack R. Wands

   Pages 111-117

10. Bovine Leukemia Virus Ribozymes

    • Glenn H. Cantor

    Pages 119-129

11. Use of Ribozymes to Specifically Alter the MDR-1 Phenotype

    • David Y. Bouffard

    Pages 131-152

12. Adenoviral-Mediated Anti-H-ras Ribozyme for the Treatment of Bladder Cancer

    • Akira Irie, ierry Tsai, Mohammed Kashani-Sabet

    Pages 153-168

13. Tissue-Specific Delivery of an anti-H-ras Ribozyme Against Malignant Melanoma

    • Tsukasa Ohkawa, Mohammed Kashani-Sabet

    Pages 169-192

14. Ribozymes as a Novel Approach for the Treatment of Human Pancreatic carcinoma

    • Hiroshi Kijima, David Y. Bouffard, Kevin J. Scanlon

    Pages 193-208

15. K-rasRibozyme for Lung Cancer

    • Alex W. Tong, Yu-An Zhang, John Nemunaitis, Gabriele Mues

    Pages 209-222

16. Anti-c-erb-B-2 Ribozyme for Breast Cancer

    • Toshiya Suzuki, Lisa D. Curcio, Jerry Tsai, Mohammed Kashani-Sabet

    Pages 223-239

17. Ribozymes

    • Gunhild M. Mælandsmo, Inge Kjønniksen, Eivind Hovig, Kevin J. Scanlon, Øystein Fodstad

    Pages 241-260

18. Preformed Ribozymes

    • Mouldy Sioud

    Pages 261-278

19. 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, E. Premkumar Reddy

    Pages 279-292

20. Ribozymes Targeted Against AML1/MTG8

    • Hiromichi Matsushita, Hiroyuki Kobayashi, Masahiro Kizaki, Yasuo Ikeda

    Pages 293-310

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.

". . . an impressive collection of ribozyme applications. . .a useful investment for anyone who contemplates or is actively using ribozymes for target RNA inactivations."-Nature Medicine

• Berlex Biosciences, Richmond

  Kevin J. Scanlon

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