Tài liệu Targets in gene therapy Edited by Yongping You

TARGETS IN GENE THERAPY
Edited by Yongping You

Contents
Preface IX


Part 1 Target Strategy in Gene Therapy 1
Chapter 1 Choosing Targets for Gene Therapy 3
Karina J. Matissek, Ruben R. Bender,
James R. Davis and Carol S. Lim
Chapter 2 Gene Modulation by Peptide Nucleic
Acids (PNAs) Targeting microRNAs (miRs) 29
Rosangela Marchelli, Roberto Corradini, Alex Manicardi,
Stefano Sforza, Tullia Tedeschi, Enrica Fabbri, Monica Borgatti,
Nicoletta Bianchi and Roberto Gambari
Chapter 3 Effective Transgene Constructs to
Enhance Gene Therapy with Trichostatin A 47
Hideki Hayashi, Yuhua Ma, Tomoko Kohno, Masayuki Igarashi,
Kiyoshi Yasui, Koon Jiew Chua,Yoshinao Kubo,
Motoki Ishibashi, Ryuji Urae, Shin Irie and Toshifumi Matsuyama
Chapter 4 Suicide Gene Therapy by Herpes
Simplex Virus-1 Thymidine Kinase (HSV-TK) 65
Dilip Dey and Gregory R.D. Evans
Chapter 5 Translational Challenges for
Hepatocyte-Directed Gene Transfer 77
Stephanie C. Gordts, Eline Van Craeyveld,
Frank Jacobs and Bart De Geest
Chapter 6 Physiologically-Regulated
Expression Vectors for Gene Therapy 99
Olivia Hibbitt and Richard Wade-Martins
Chapter 7 PLP-Dependent Enzymes: a Potent
Therapeutic Approach for Cancer
and Cardiovascular Diseases 119
Ashraf S. El-Sayed and Ahmed A. Shindia
Chapter 8 Improvement of FasL Gene Therapy In Vitro
by Fusing the FasL to Del1 Protein Domains 147
Hisataka Kitano, Atsushi Mamiya and Chiaki Hidai
Chapter 9 Feasibility of BMP-2 Gene Therapy
Using an Ultra-Fine Needle 159
Kenji Osawa, Yasunori Okubo, Kazumasa Nakao,
Noriaki Koyama and Kazuhisa Bessho


Part 2 Gene Therapy of Cancer 167
Chapter 10 Current Strategies for Cancer Gene Therapy 169
Yufang Zuo, Xiaofang Ying, Hui Wang,
Wen Ye, Xiangqi Meng, Hongyan Yu,
Yi Zhou, Wuguo Deng and Wenlin Huang
Chapter 11 Gene Therapy Strategy for Tumour Hypoxia 185
Hiroshi Harada
Chapter 12 Gene Therapy of Glioblastoma:
Anti – Gene Anti IGF-I Strategy 201
Jerzy Trojan
Chapter 13 Mechanism of Hypoxia-Inducible Factor-1alpha
Over- Expression and Molecular-Target
Therapy for Hepatocellular Carcinoma 225
Dengfu Yao, Min Yao, Shanshan Li and Zhizhen Dong
Chapter 14 Cancer Gene Therapy
via NKG2D and FAS Pathways 243
Yanzhang Wei, Jinhua Li and Hari Shankar R. Kotturi
Chapter 15 Emergence of IFN-lambda
as a Potential Antitumor Agent 275
Ahmed Lasfar and Karine A. Cohen-Solal
Chapter 16 Intramuscular IL-12 Electrogene Therapy
for Treatment of Spontaneous Canine Tumors 299
Maja Cemazar, Gregor Sersa,
Darja Pavlin and Natasa Tozon

Part 3 Gene Therapy of Other Diseases 321
Chapter 17 Gene Therapy Targets and the Role of
Pharmacogenomics in Heart Failure 323
Dimosthenis Lykouras, Christodoulos Flordellis
and Dimitrios Dougenis
Chapter 18 Gene Therapy of the Heart through
Targeting Non-Cardiac Cells 337
Guro Valen
Chapter 19 Transplantation of Sendai Viral
Angiopoietin-1-Modified Mesenchymal
Stem Cells for Ischemic Heart Disease 357
Jianhua Huang, Huishan Wang and Hirofumi Hamada
Chapter 20 Using Factor VII in Hemophilia Gene Therapy 369
Bahram Kazemi
Chapter 21 The Different Effects of TGF-β1, VEGF and
PDGF on the Remodeling of Anterior
Cruciate Ligament Graft 389
Changlong Yu, Lin Lin and Xuelei Wei
Chapter 22 Different ex Vivo and Direct in Vivo DNA
Administration Strategies for Growth
Hormone Gene Therapy in Dwarf Animals 396
Cibele Nunes Peroni, Nélio Alessandro de Jesus Oliveira,
Claudia Regina Cecchi, Eliza Higuti and Paolo Bartolini
Chapter 23 Protection from Lethal Cell Death in Cecal
Ligation and Puncture-Induced Sepsis Mouse
Model by In Vivo Delivery of FADD siRNA 409
Yuichi Hattori and Naoyuki Matsuda
Chapter 24 Muscle-Targeted Gene Therapy
of Charcot Marie-Tooth Disease
is Dependent on Muscle Activity 423
Stephan Klossner, Marie-Noëlle Giraud,
Sara Sancho Oliver, David Vaughan and Martin Flück


Preface
Up to now, major diseases often attempted to be treated by gene therapy include
cancer, cardiovascular disease and monogenic diseases. Despite many decades of gene
therapy research on these fatal diseases, most of the products fail to make it to market.
One urgent problem is to identify the key targets for specific drugs.
The aim of our book is to cover key aspects of existing problems in the emerging field
of targets in gene therapy. With the contribution of leading experts and pioneers in
various disciplines of gene therapy, this book brings together major approaches:
1. Target Strategy in Gene Therapy,
2. Gene Therapy of Cancer,
3. Gene Therapy of Other Diseases.
The publication of this book was made possible by the efforts and collaboration of
many individuals. We thank the contributors and section editors for generously
sharing their expertise and scientific skills.
We hope that this book will provide a realistic image of the huge potential, perspective
and challenges facing the field of gene therapy in its quest to cure disease and prolong
life.
Yongping You
Professor and Chief Physician
Department of Neurosurgery
The First Affiliated Hospital of Nanjing Medical University
China