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Gene Therapy: Therapeutic Mechanisms and Strategies

  •  ISBN-10 0-8247-7665-8

  •  ISBN-13 978-0-8247-7665-7

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publication info   N.S. Templeton and D.D. Lasic, ed., Marcel Dekker, 2000, 608 pp., hard cover
mfr. no.   Marcel Dekker

Description

General description

This reference covers all aspects of gene therapy, including viral and nonviral delivery systems, targeted diseases, regulatory issues, and FDA requirements. It investigates genetic bullets to block HIV-1 replication and genetic guns to deliver antiviral agents. It examines donor cells, vector systems, and functional genes; defines extra- and intracellular pathways and barriers for gene delivery; addresses the interaction of micelles and liposomes with nucleic acids; describes the formulations, pharmacology, and toxicology of oligonucleotides; considers RNA catalysis and decoy RNAs; and outlines hematopoietic stem cell procurement.

Table of Contents

Viral Delivery and Therapeutic Strategies
Retroviral Vectors for Gene Therapy
Paula M. Cannon and W. French Anderson
Adenovirus Vectors for Gene Therapy
Neil R. Hackett and Ronald G. Crystal
Adeno-Associated Virus and Adeno-Associated Virus Vectors for Gene Delivery
Barrie J. Carter
Ex Vivo Gene Therapy Using Myoblasts and Regulatable Retroviral Vectors
Clare R. Ozawa, Matthew L. Springer, and Helen M. Blau
Design and Use of Herpes Simplex Viral Vectors for Gene Therapy
Darren Wolfe, William F. Goins, David J. Fink, and Joseph C. Glorioso, III
Alphavirus-Based Vectors for Vaccine and Gene Therapy Applications
Thomas W. Dubensky, Jr., John M. Polo, and Douglas J. Jolly

Nonviral Delivery and Therapeutic Strategies
Gene Delivery with Polyethylenimine
S. M. Zou, Jean-Paul Behr, D. Goula, and Barbara Demeneix
Receptor-Mediated Gene Transfer
Kurosh Ameri and Ernst Wagner
Modification of Melanoma Cells via Ballistic Gene Delivery for Vaccination
Ulrich R. Hengge and Dirk Schadendorf
Polymer-Encapsulated Cells for Gene Therapy
Dwaine F. Emerich and Elizabeth Razee
Molecular Interactions in Lipids, DNA, and DNA-Lipid Complexes
Rudolf Podgornik, Helmut H. Strey, and V. A. Parsegian
Bioorganic Colloids: Macromolecules, DNA, Self-Assembled Particles, and Their Complexes
Danilo D. Lasic and Nancy Smyth Templeton
Synthetic Virus Systems for Systemic Gene Therapy
Ian MacLachlan, Pieter Cullis, and Roger W. Graham

Other Therapeutic Strategies and Regulatory Aspects
Gene Therapy Applications of Ribozymes
Bruce A. Sullenger and Lynn Milich
Antisense Oligonucleotide–Based Therapeutics
C. Frank Bennett, Madeline Butler, P. Dan Cook, Richard S. Geary, Arthur A. Levin, Rahul Mehta, Ching-Leou Teng, Hemant Deshmukh, Lloyd Tillman, and Greg Hardee
Selectable Markers for Gene Therapy
Michael M. Gottesman, Thomas Licht, Yi Zhou, Caroline Lee, Tzipora Shoshani-Kupitz, Peter Hafkemeyer, Christine A. Hrycyna, and Ira Pastan
Suicide Gene Therapy
Bert W. O’Malley, Jr.
Regulatory Aspects of Gene Therapy
Andra E. Miller and Stephanie L. Simek

Disease Targets and Therapeutic Strategies
Gene Transfer into Hematopoietic Stem Cells
David M. Bodine
Gene Therapy for Hematopoietic Disorders
Mitchell E. Horwitz and Harry L. Malech
Gene Therapy for Cardiovascular Disease and Vascular Grafts
Afshin Ehsan, Michael J. Mann, and Victor J. Dzau
Gene Therapy for Cancer
Karsten Brand, Gerhard Wolff, and Michael Strauss
Barriers to Efficient Airway Epithelial Gene Transfer: Lessons from Cystic Fibrosis
Larry G. Johnson and Richard C. Boucher
Gene Transfer to the Central Nervous System
Ulrike Blömer, I. M. Verma, and F. H. Gage
Principles of Gene Therapy for Inborn Errors of Metabolism
Jon A. Wolff and Cary O. Harding
Gene Therapy and HIV-1 Infection
Ralph Dornburg and Roger J. Pomerantz
Gene Delivery to the Skin
Paul A. Khavari
DNA Vaccines
Jong J. Kim and David B. Weiner

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