Clinical Trials of Genetic Therapy with Antisense DNA and DNA Vectors

by Eric Wickstrom (Editor)

Overview

An important new collection of clinical and preclinical reports on genetic therapy, this book describes illustrative examples of diseases in which gene-based interventions are presently plausible, and presents case studies of current research using both synthetic oligonucleotides and biological vectors.
Combining the insights of over 50 contributors, Clinical Trials of Genetic Therapy with Antisense DNA and DNA Vectors

furnishes a historical overview of genetic therapy

highlights official Food and Drug Administration positions on the preparation of oligonucleotides and vectors

offers practical models of agent preparation, animal testing, pharmacokinetics, toxicology, and clinical trials

discusses both synthetic DNA and biological vector approaches to cancer, viral, and cardiological indications

illustrates for new practitioners how each stage of genetic therapy is developed

details genetic treatment of leukemia; lymphoma; cancer of the brain, breast, colon, kidney, and lung; melanoma; HIV; and coronary restenosis

includes examples of antisense, ribozyme, tumor suppressor, immunostimulation, and gene replacement therapy

and addresses questions of preparation, delivery, toxicity, mechanism, and specificity.

Product Details

ISBN-13:	9781000145953
Publisher:	CRC Press
Publication date:	08/13/2020
Sold by:	Barnes & Noble
Format:	eBook
Pages:	448
File size:	8 MB

About the Author

Eric Wickstrom (Edited by)

Table of Contents

A Brief History of Genetic Therapy: Gene Therapy, Antisense Technology, and Genomics, James W. Hawkins Preclinical Development of Oligonucleotide Therapeutics for Cancer: Regulatory Aspects, Chang-Ho Ahn and Joseph J. DeGeorge Commercial Scale Manufacturing of Oligonucleotides Under Good Manufacturing Practices, Jose E. Gonzalez, Richard G. Einig, Patricia Puma, Timothy P.Noonan, Paul E. Kennedy, Bruce G. Sturgeon, Bing H. Wang, and Jin-yan Tang The Regulatory Process and Gene Therapy, Suzanne L. Epstein Production of Clinical Lots of Gene Therapy Vectors Using Good Manufacturing Practice: Experience in a University Setting, Alan R. Davis and Colleen Baker Gene Therapy Clinical Trials for Adenosine Deaminase Deficiency/Severe Combined Immunodeficiency, Erlinda M. Gordon and W. French Anderson Development of an Oligodeoxynucleotide Pharmaceutical for the Treatment of Human Leukemia, Alan M. Gewirtz and Deborah Lee Sokol Clinical Trials with Anti-p53 DNA, OL(1)p53, in Patients with Acute Myelogenous Leukemia and Myelodysplastic Syndrome, Patrick L. Iversen Human Bcl-2 Antisense Therapy for Lymphomas, Finbarr E. Cotter, Andrew Webb, Paul Clarke, and David Cunningham Retroviral Gene Transfer in Autologous Bone Marrow and Stem Cell Transplantation, Rafat Abonour and Kenneth Cornetta Adenoviral Gene Transfer of the Herpes Virus Thymidine Kinase Gene for Treating Gliomas, Jane B. Alavi, Jason G. Smith, and Stephen L. Eck Distribution and Toxicity of Retroviral Vectors After Intracavitary Delivery in Mouse and Man, Patrice S. Obermiller, Anne M. Pilaro, Carlos L. Arteaga, and Jeffrey T. Holt Clinical and Immunologic Responses to Gene Transfer of an Allogeneic Major Histocompatibility Complex Antigen, Alison T. Stopeck and Evan M. Hersh Defective Tumor Suppressor Gene Replacement and Oncogene Inactivation for the Treatment of Cancer, Jack Roth The Molecular Basis of Bladder Cancer and Prospects for Gene Therapy Using Hammerhead Ribozymes, Eric J. Small, Mohammed Kashani-Sabet, David Y. Bouffard, and Kevin J. Scanlon In Situ Gene Insertion for Immunotherapy Using Vaccinia Virus Vectors, Edmund C. Lattime, Laurence C. Eisenlohr, and Michael J. Mastrangelo Antisense Oligonucleotide-Based Therapy for HIV-1 Infection from Laboratory to Clinical Trials, Sudhir Agrawal Treatment of Retinitis Induced by Cytomegalovirus Using Intravitreal Fomivirsen (Isis 2922), Stanley T. Crooke Synthetic DNA-Based Compounds for the Prevention of Coronary Restenosis: Current Status and Future Challenges, Andrew Zalewski, Yi Shi, John D. Mannion, and Fernando Roqué Prevention of Restenosis by Gene Targeting, Michael J. Mann, Heiko E. Von der Leyen, and Victor J. Dzau

Editorial Reviews

This is a collection of studies using gene-based interventions to treat human disease. This anthology gives the reader a review of experimental evidence demonstrating that state-of-the-art gene-based technology will be the mainstay for treating human diseases. It is written for experienced clinical and biomedical investigators in the field of biological sciences and medicine. However, it is heavily laden with DNA-based therapies on hematological malignancies and solid tumors and therefore may be less applicable to audiences outside these areas. It provides the reader with an historical perspective of gene therapy and insight into the future of this technology. Each chapter highlights the latest research in gene targeting and manipulation and its usefulness as a clinical tool once perfected. Translating science from the laboratory bench to the patient in bed is an effective teaching tool. A nice addition in this mostly research-based text is the chapter covering the perspective of the Food and Drug Administration towards the use of adenovirus vectors and how this agency plans to regulate product development. The black-and-white illustrations are adequate and demonstrate the point the authors are trying to make, but they are not especially visually appealing. There is no glossary, which is a major shortcoming, but there is a very thorough reference listing after each chapter. Although this book has a few weaknesses, it is a useful resource for basic science and clinical investigators who are interested in learning the current status of research in gene-based technology and its future clinical application. However, as with everything in science, what is reported today is oftenoutdated by the time it reaches the bookshelves. Readers will gain an understanding of the usefulness of antisense DNA and vectors but should not go away with the feeling that they know the current state of affairs in this area due to its dynamic nature.

Shelly A. Cummings

This volume clearly demonstrates that genetic therapy is underway, and that the efforts described are soundly based in rationale and feasibility...Our increasing understanding of the mechanisms of conversion of the genetic code into function indicate that gene based therapy will become a mainstay of disease control in the 21st century.

O. Michael Colvin

"
This volume clearly demonstrates that genetic therapy is underway, and that the efforts described are soundly based in rationale and feasibility…Our increasing understanding of the mechanisms of conversion of the genetic code into function indicate that gene based therapy will become a mainstay of disease control in the 21st century.
"-O. Michael Colvin, Duke University Medical Center, Durham, North Carolina

From the Publisher