Clinical Trials of Genetic Therapy with Antisense DNA and DNA Vectors: 1st Edition (Hardback) book cover

Clinical Trials of Genetic Therapy with Antisense DNA and DNA Vectors

1st Edition

Edited by Eric Wickstrom

CRC Press

448 pages

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Hardback: 9780824700850
pub: 1998-04-21
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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.
  • Reviews


    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

    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

    Subject Categories

    BISAC Subject Codes/Headings:
    MEDICAL / Pharmacology
    MEDICAL / Genetics
    SCIENCE / Life Sciences / Biology / Molecular Biology