Gene and Cell Therapy: Therapeutic Mechanisms and Strategies, Fourth Edition, 4th Edition (Hardback) book cover

Gene and Cell Therapy

Therapeutic Mechanisms and Strategies, Fourth Edition, 4th Edition

Edited by Nancy Smyth Templeton

CRC Press

1,337 pages | 69 Color Illus. | 301 B/W Illus.

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Hardback: 9781466571990
pub: 2015-01-20
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The Most Comprehensive, State-of-the-Art Book on Using Gene and Cell Therapy in Clinical Medicine

Gene and Cell Therapy: Therapeutic Mechanisms and Strategies, Fourth Edition presents extensive background and basic information, state-of-the-art technologies, important achievements, and lingering challenges in the fields of gene and cell therapies. The fourth edition of this bestseller continues to provide the most comprehensive coverage of these fields in one volume. Some chapters have expanded introductions, making the book even more suitable for classroom use. This edition also offers more material on the contributors’ research efforts as well as current technologies, disease targets, and clinical applications.

Divided into four sections, the book covers:

  • Delivery systems and therapeutic strategies
  • Other therapeutic strategies, including technologies that knock down gene expression
  • Gene expression, regulation, and detection
  • Gene and cell therapies, disease targets, clinical trials, and regulatory issues

Accessible to a broad audience, including students, scientists, physicians, and lay people, this book provides readers with up-to-date, interdisciplinary knowledge and tools to tackle the evolving areas of gene therapy, cell therapy, and tissue engineering. From introductory information to state-of-the-art technologies and concepts, the book helps readers understand vector design and construction, delivery systems, therapeutic strategies, gene expression and detection, disease targets, clinical applications and trials, cell-based therapies, novel imaging systems, gene regulation, and regulatory affairs.


"This textbook edited by Nancy Smyth Templeton is exactly what is needed to advance the fields of gene and cell therapy and for general education and further refinement of the technologies. … With over 1,000 pages describing the current gene transfer and vector technologies and the basics of cell therapy and clinical applications, this fourth edition of Gene and Cell Therapy: Therapeutic Mechanisms and Strategies will give an excellent, comprehensive basis to advance gene medicine and cell therapy toward successful future applications in medical practice."

—From the Foreword by Seppo Ylä-Herttuala, MD, PhD, FESC, Past President, European Society of Gene and Cell Therapy, and Professor of Molecular Medicine, University of Eastern Finland

"The newly updated edition contains additional chapters covering promising gene and cell therapy clinical trials…. The book is also suitable for readers from all backgrounds including students, physicians, scientists, and other individuals interested in understanding the current status of gene and cell therapies and their contribution to the field of medicine.", February 2015

Table of Contents

Delivery Systems and Therapeutic Strategies

Lentiviral Vectors: Design and Applications Anais Girard and Els Verhoeyen

Adenovirus-Vector-Mediated Gene Delivery: Barriers and Chemical Vector Modifications Florian Kreppel

Helper-Dependent Adenoviral Vectors for Gene Therapy Nicola Brunetti-Pierri and Philip Ng

Adeno-Associated Virus and AAV Vectors for Gene Delivery Barrie J. Carter

Vaccinia Viral Vectors for Vaccines and Oncolytic Virotherapy Z. Sheng Guo, J. Andrea McCart, and David L. Bartlett

Oncolytic Measles Virus-Based Delivery Systems Pavlos Msaouel and Evanthia Galanis

Alphavirus Vectors in Gene Therapy and Neuroscience Kenneth Lundstrom

Oncolytic Viruses for Treatment of Cancer Akseli Hemminki, Markus Vähä-Koskela, and Vincenzo Cerullo

Baculovirus: A High-Capacity Vector for Versatile Gene Transfer

Kari J. Airenne and Seppo Ylä-Herttuala

Baculovirus as a Gene Delivery Vector for Bone and Cartilage Tissue Engineering Chin-Yu Lin, Chiu-Ling Chen, Li-Yu Sung, Kuei-Chang Li, Chia-Lin Yeh, and Yu-Chen Hu

Bacteria as Gene Therapy Vectors for Cancer William L. Byrne and Mark Tangney

Redox-Responsive Polymer-Based Gene Delivery Systems Yu Zhu, Jing Li, Yan Wang, Devika Soundara Manickam, Yezi You, and David Oupický

Receptor-Targeted Polyplexes for pDNA and siRNA Delivery Linda Beckert, Alexander Philipp, and Ernst Wagner

Dendrimers, Dendrigrafts, and Their Conjugations as Delivery Vectors in Gene Therapy Ampornphan Siriviriyanun and Toyoko Imae

Gene Electrotransfer Shawna A. Shirley, Loree C. Heller, and Richard Heller

Magnetofection: Using Magnetic Particles and Magnetic Force to Enhance and Target Nucleic Acid Delivery Christian Plank, Dialechti Vlaskou, Yolanda Sanchez-Antequera, and Olga Mykhaylyk

New Advances in Cell-Based Drug and Gene Delivery Systems José M. Lanao, Carmen Gutiérrez Millán, and Clara Isabel Colino Gandarillas

Pressurized Transvenous-Retrograde Extremity Perfusion Zheng Fan, William Powers, Joe Kornegay, and Xiao Xiao

Optimization of Nonviral Gene Therapeutics Using Bilamellar Invaginated Vesicles Nancy Smyth Templeton

Other Therapeutic Strategies

Antisense Oligonucleotide–Based Therapeutics C. Frank Bennett, Eric Swayze, Scott Henry, and Richard Geary

Development of RNAi Therapeutics Akin Akinc, Brian R. Bettencourt, and Martin A. Maier

Therapeutic Applications of Morpholino Oligonucleotides David R. Soto-Pantoja, Jeffrey S. Isenberg, and David D. Roberts

Therapeutic Potential of microRNAs Tina Catela Ivkovic and George A. Calin

Gene Expression, Regulation, and Detection; Genome Modifications

Monitoring Gene and Cell Therapies in Living Subjects with Molecular Imaging Technologies John Ronald, Sandip Biswal, and Sanjiv Sam Gambhir

Deliberate Regulation of Therapeutic Transgenes: Recent Advances in System Development and Uses Nuria Vilaboa and Richard Voellmy

Nonviral Genome Modification Strategies for Gene Therapy: Transposon, Integrase, and Nuclease Systems Lauren E. Woodard and Michele P. Calos

Selectable Markers for Gene Therapy Jean-Pierre Gillet, Ryan C. Hunt, Chava Kimchi-Sarfaty, Tal Kafri, Jizhong Zou, Mahendra S. Rao, and Michael M. Gottesman

Gene and Cell Therapies, Disease Targets, Clinical Trials, and Regulatory Issues

Pluripotent Stem Cells and Their Therapeutic Applications Sadaf Amin and Shuibing Chen

Adult Lung Stem Cells Amritha Kidiyoor, Anthony Atala, and Sean V. Murphy

Placenta-Derived Cells and Their Therapeutic Applications De Debashree, Gabriela Kmiecik, Anna Cargnoni, and Ornella Parolini

Directing Pluripotent Human Embryonic Stem Cells toward Lineage-Specific Cell Therapy Derivatives for Regenerative Medicine Xuejun H. Parsons

Surgical and Translational Aspects of Tissue Engineering Peggy Lange, Jonathan M. Fishman, Paolo De Coppi, and Martin A. Birchall

DNA Vaccines Jian Yan and David B. Weiner

Cell and Gene Therapies for Cardiovascular Disease Michael G. Katz, Anthony S. Fargnoli, Richard D. Williams, and Charles R. Bridges

Gene Therapy for Hematopoietic Disorders Elizabeth M. Kang and Harry L. Malech

Update on Preclinical and Clinical Gene Therapy for the Treatment of Cystic Fibrosis Uta Griesenbach and Eric Alton

Gene Therapy for Vision Disorders Deniz Dalkara and José-Alain Sahel

Cell and Gene Therapies for the Treatment of Hearing Disorders Lisa N. Gillespie, Rachael T. Richardson, Andrew K. Wise, and Bryony A. Nayagam

Molecular Therapy for Type 1 and Type 2 Diabetes Susan Leanne Samson, Vijay Yechoor, and Lawrence Chan

Alipogene Tiparvovec: Gene Therapy for Familial Lipoprotein Lipase Deficiency Norman E. Miller

Gene and Cell Therapies for Muscular Dystrophies Sara Benedetti, Giulio Cossu, and Francesco Saverio Tedesco

Gene Therapy Clinical Trials for Muscular Dystrophy Louise R. Rodino-Klapac and Jerry R. Mendell

RNA Interference-Based Gene Therapy Strategies for the Treatment of HIV Infection Lisa J. Scherer, John C. Burnett, and John J. Rossi

Gene Therapy for the Treatment of Cancer: From Laboratory to Bedside Carlos A. Ramos, Barbara Savoldo, and Gianpietro Dotti

Gene Therapy for the Treatment of Lung Cancer Humberto Lara-Guerra and Jack Roth

FANG: bi-shRNAifurin and GMCSF DNA-Augmented Autologous Tumor Cell Vaccine: Clinical Results John Nemunaitis, Donald D. Rao, and Neil Senzer

Cutaneous Cell- and Gene-Based Therapies for Inherited and Acquired Skin Disorders Jean Christopher Chamcheu, Vaqar M. Adhami, Imtiaz A. Siddiqui, and Hasan Mukhtar

Cell Therapy for the Creation of Cartilage and Related Clinical Trials Jonathan Karnes, Ying Zhang, and Ming Pei

Cell Therapies for the Treatment of Inborn Metabolic Errors María José Gómez-Lechón, Laia Tolosa, Eugenia Pareja, and José V. Castell

Cell Therapies for Lung Disease Rebecca Lim, Jean L. Tan, and Euan M. Wallace

Gene Therapy for Neurological Diseases Massimo S. Fiandaca, Shreyas Mahapatra, Suresh de Silva, William J. Bowers, and Howard J. Federoff

Gene Therapy for Alzheimer’s Disease Alan H. Nagahara and Mark H. Tuszynski

Translating Stem Cell Therapy for Alzheimer’s Disease Kazutaka Shinozuka, Paolina Pantcheva, Sandra Acosta, Mibel Pabon, Naoki Tajiri, Yuji Kaneko, and Cesar V. Borlongan

Regulatory Aspects of Gene Therapy Daniel Takefman and Lilia Bi


About the Editor

Nancy Smyth Templeton earned her PhD in molecular biology and biochemistry from Wesleyan University. She underwent postdoctoral training at the National Institutes of Health in the NCI and the NHLBI. She was recently the director of delivery systems at Gradalis/Strike Bio Inc., focusing on targeted delivery of bifunctional shRNA therapeutics to solid tumors by intravenous injections using her robust nanoparticles: bilamellar invaginated vesicles (BIVs). Dr. Templeton has published 62 peer-reviewed papers, presented over 170 seminars, been issued 16 patents, and won several awards.

Subject Categories

BISAC Subject Codes/Headings:
SCIENCE / Life Sciences / Cytology
SCIENCE / Life Sciences / Genetics & Genomics
SCIENCE / Life Sciences / Biology / Molecular Biology