Therapeutic Applications of Ribozymes
Therapeutic Applications of Ribozymes

Author: Kevin J. Scanlon

Publisher: Springer Science & Business Media

Published: 1998-06-23

Total Pages: 488

ISBN-13:

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In Therapeutic Applications of Ribozymes, expert laboratory scientists describe in detail their methodologies for constructing ribozymes designed to elucidate the role of specific genes as key routes to the development of novel therapies for a wide variety of diseases. The authors review the many sites targeted with ribozymes in various diseases and provide specific accounts of the practical techniques required for the proper use of ribozymes in these systems. Their cutting-edge protocols demonstrate how to achieve ribozyme expression in distinct cellular systems, the preparation and use of high-efficiency ribozyme DNA or RNA delivery, and the studies required to prove the efficacy of ribozyme-mediated inhibition, with its concomitant effect on phenotypic parameters. Therapeutic Applications of Ribozymes contains all the practical detail necessary to realize the high promise of ribozyme technology as a significant, widely used methodology. Focused on ribozyme targets and the novel associated technology, the book immediately becomes the leading resource for all those seeking to transform this new category of therapeutic agent into efficacious clinical practice.

Therapeutic Applications of Ribozymes and Riboswitches
Therapeutic Applications of Ribozymes and Riboswitches

Author: Daniel A Lafontaine

Publisher: Humana Press

Published: 2013-12-07

Total Pages: 0

ISBN-13: 9781627037297

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In Therapeutic Applications of Ribozymes and Riboswitches: Methods and Protocols, expert researchers in the field provide a complete overview of protocols used in the development of RNA molecule as drugs and drug target. These include methods and protocols on recent and precise RNA molecule approaches or studies in the development of an RNA therapeutic tool. We are convinced that these methods will help researchers from various domains of life sciences, including clinicians, biochemists and virologists. Written in the highly successful Methods in Molecular Biology series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and key tips on troubleshooting and avoiding known pitfalls. Authoritative and practical, Therapeutic Applications of Ribozymes and Riboswitches: Methods and Protocols seeks to aid scientists in the further development of new therapeutic applications implicating RNA molecules or directly targeting harmful RNA.

Ribozymes
Ribozymes

Author: Sabine Müller

Publisher: John Wiley & Sons

Published: 2021-07-09

Total Pages: 81

ISBN-13: 3527814531

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Ribozymes Provides comprehensive coverage of a core field in the molecular biosciences, bringing together decades of knowledge from the world’s top professionals in the field Timely and unique in its breadth of content, this all-encompassing and authoritative reference on ribozymes documents the great diversity of nucleic acid-based catalysis. It integrates the knowledge gained over the past 35 years in the field and features contributions from virtually every leading expert on the subject. Ribozymes is organized into six major parts. It starts by describing general principles and strategies of nucleic acid catalysis. It then introduces naturally occurring ribozymes and includes the search for new catalytic motifs or novel genomic locations of known motifs. Next, it covers the development and design of engineered ribozymes, before moving on to DNAzymes as a close relative of ribozymes. The next part examines the use of ribozymes for medicinal and environmental diagnostics, as well as for therapeutic tools. It finishes with a look at the tools and methods in ribozyme research, including the techniques and assays for structural and functional characterization of nucleic acid catalysts. The first reference to tie together all aspects of the multi-faceted field of ribozymes Features more than 30 comprehensive chapters in two volumes Covers the chemical principles of RNA catalysis; naturally occurring ribozymes, engineered ribozymes; DNAzymes; ribozymes as tools in diagnostics and therapy, and tools and methods to study ribozymes Includes first-hand accounts of concepts, techniques, and applications by a team of top international experts from leading academic institutions Dedicates half of its content to methods and practical applications, ranging from bioanalytical tools to medical diagnostics to therapeutics Ribozymes is an unmatched resource for all biochemists, biotechnologists, molecular biologists, and bioengineers interested in the topic.

Ribozymes and RNA Catalysis
Ribozymes and RNA Catalysis

Author: David Malcolm James Lilley

Publisher: Royal Society of Chemistry

Published: 2008

Total Pages: 339

ISBN-13: 0854042539

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Takes the reader through the origins of catalysis in RNA and necessarily includes significant discussion of structure and folding. The main focus of the book concerns chemical mechanism with extensive comment on how, despite the importance of RNA catalysis in the cell, its origins are still poorly understood and often controversial. The reader is given an outline of the important role of RNA catalysis in many aspects of cell function, including RNA processing and translation.

Ribozymes and siRNA protocols
Ribozymes and siRNA protocols

Author: Mouldy Sioud

Publisher: Humana Press

Published: 2004-03-05

Total Pages: 624

ISBN-13: 9781588292261

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In this completely updated and expanded edition of a classic bench manual, hands-on experts take advantage of the latest advances in ribozyme, DNAzyme, and RNA interference technologies to describe in detail the exciting and successful methods now available for gene inactivation in vitro and in vivo. Their optimized techniques employ hairpain ribozymes, DNAzymes, hammerhead ribozymes and derivatives, group I intron ribozymes, Rnase P ribozymes, and siRNAs, as well as general methods for RNA structure analysis, delivery of oligonucleotides, and gene therapy. Also provided are novel methods for identifying accessible cellular mRNA sites; group I intron and RNAse P ribozymes protocols for effective design, selection, and therapeutic applications; and the latest RNAi methods for sequencing-specific gene silencing in a wide variety of organisms. Comprehensive and up-to-date, Ribozymes and siRNA Protocols synthesizes for experienced and novice investigators alike the exciting advances in understanding nucleic acid enzymes and demonstrates how they may be used to analyze gene function and target validation, and to productively develop new therapeutics for human diseases.

Therapeutic Oligonucleotides
Therapeutic Oligonucleotides

Author: Jens Kurreck

Publisher: Royal Society of Chemistry

Published: 2008

Total Pages: 362

ISBN-13: 0854041168

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This book provides a compelling overall update on current status of RNA interference

RNA Therapeutics
RNA Therapeutics

Author: Mouldy Sioud

Publisher: Humana Press

Published: 2011-03-04

Total Pages: 527

ISBN-13: 9781607616641

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Central to the synthesis of proteins, the performance of catalysis, and many other physiological processes, the aberrant expression of which can be linked to human diseases including cancers, RNA has proven to be key target for therapeutics as well as a tool for therapy. In RNA Therapeutics: Function, Design, and Delivery, expert contributors from a broad spectrum of scientific backgrounds highlight the roles that messenger RNAs and small RNAs can play in biology and medicine. While covering the five major RNA-based drugs, namely the use of ribozymes to cleave and/or correct mRNA transcript, the use of siRNA for targeted silencing of gene transcripts, the use of aptamers, like short RNA molecules, for neutralizing the protein functions, the use mRNA-transfected DCs to activate immune system against tumor cells, as well as the use of RNA to reprogram T and/or DC cell function, this extensive volume brings together the fields of coding (mRNA) and non-coding RNA such as ribozymes, RNAse P, siRNAs, and miRNAs into one convenient source. Written in the highly successful Methods in Molecular BiologyTM series format, the cutting-edge protocol chapters contain introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and practical tips on troubleshooting and avoiding known pitfalls. Also, the book contains several excellent reviews for teaching purposes. Authoritative and comprehensive, RNA Therapeutics: Function, Design, and Delivery provides key models and tools which will assist researchers in increasing our understanding of RNA functions, modifications, and their involvement in diseases in order to lead to the design of vital new RNA-based therapeutics.

Antisense and Ribozyme Methodology
Antisense and Ribozyme Methodology

Author: Ian Gibson

Publisher: John Wiley & Sons

Published: 2008-09-26

Total Pages: 92

ISBN-13: 3527612521

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Antisense and ribozymes have a relatively short yet successful history as research tools in gene expression studies, and thus are considered as having high potential reagents in treating viral infections and cancer. This laboratory companion provides detailed information on the potential, advantages and limitations of this methodology. It critically discusses potential pitfalls, presents strategies for choosing targets and delivery systems, so as to allow the selection of the optimum methodology for achieving fast and reliable experimental success with any human or other biological system. For researchers, technicians and advanced graduates in experimental medicine, molecular and cell biology.

Immunopharmacology
Immunopharmacology

Author: Manzoor M. Khan

Publisher: Springer Science & Business Media

Published: 2008-12-19

Total Pages: 275

ISBN-13: 0387779760

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During the past decades, with the introduction of the recombinant DNA, hybridoma and transgenic technologies there has been an exponential evolution in understanding the pathogenesis, diagnosis and treatment of a large number of human diseases. The technologies are evident with the development of cytokines and monoclonal antibodies as therapeutic agents and the techniques used in gene therapy. Immunopharmacology is that area of biomedical sciences where immunology, pharmacology and pathology overlap. It concerns the pharmacological approach to the immune response in physiological as well as pathological events. This goals and objectives of this textbook are to emphasize the developments in immunology and pharmacology as they relate to the modulation of immune response. The information includes the pharmacology of cytokines, monoclonal antibodies, mechanism of action of immune-suppressive agents and their relevance in tissue transplantation, therapeutic strategies for the treatment of AIDS and the techniques employed in gene therapy. The book is intended for health care professional students and graduate students in pharmacology and immunology.

A Guide to Human Gene Therapy
A Guide to Human Gene Therapy

Author: Roland W. Herzog

Publisher: World Scientific

Published: 2010

Total Pages: 415

ISBN-13: 9814280917

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1. Non-viral gene therapy / Sean M. Sullivan -- 2. Adenoviral vectors / Stuart A. Nicklin and Andrew H. Baker -- 3. Retroviral vectors and integration analysis / Cynthia C. Bartholomae [und weitere] -- 4. Lentiviral vectors / Janka Matrai, Marinee K.L. Chuah and Thierry VandenDriessche -- 5. Herpes simplex virus vectors / William F. Goins [und weitere] -- 6. Adeno-Associated Viral (AAV) vectors / Nicholas Muzyczka -- 7. Regulatory RNA in gene therapy / Alfred. S. Lewin -- 8. DNA integrating vectors (Transposon, Integrase) / Lauren E. Woodard and Michele P. Calos -- 9. Homologous recombination and targeted gene modification for gene therapy / Matthew Porteus -- 10. Gene switches for pre-clinical studies in gene therapy / Caroline Le Guiner [und weitere] -- 11. Gene therapy for central nervous system disorders / Deborah Young and Patricia A. Lawlor -- 12. Gene therapy of hemoglobinopathies / Angela E. Rivers and Arun Srivastava -- 13. Gene therapy for primary immunodeficiencies / Aisha Sauer, Barbara Cassani and Alessandro Aiuti -- 14. Gene therapy for hemophilia / David Markusic, Babak Moghimi and Roland Herzog -- 15. Gene therapy for obesity and diabetes / Sergei Zolotukhin and Clive H. Wasserfall -- 16. Gene therapy for Duchenne muscular dystrophy / Takashi Okada and Shin'ichi Takeda -- 17. Cancer gene therapy / Kirsten A.K. Weigel-Van Aken -- 18. Gene therapy for autoimmune disorders / Daniel F. Gaddy, Melanie A. Ruffner and Paul D. Robbins -- 19. Gene therapy for inherited metabolic storage diseases / Cathryn Mah -- 20. Retinal diseases / Shannon E. Boye, Sanford L. Boye and William W. Hauswirth -- 21. A brief guide to gene therapy treatments for pulmonary diseases / Ashley T. Martino, Christian Mueller and Terence R. Flotte -- 22. Cardiovascular disease / Darin J. Falk, Cathryn S. Mah and Barry J. Byrne