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GENE THERAPY APPLICATIONS Edited by Chunsheng Kang Gene Therapy Applications Edited by Chunsheng Kang Published by InTech Janeza Trdine 9, 51000 Rijeka, Croatia Copyright © 2011 InTech All chapters are Open Access articles distributed under the Creative Commons Non Commercial Share Alike Attribution 3.0 license, which permits to copy, distribute, transmit, and adapt the work in any medium, so long as the original work is properly cited. After this work has been published by InTech, authors have the right to republish it, in whole or part, in any publication of which they are the author, and to make other personal use of the work. Any republication, referencing or personal use of the work must explicitly identify the original source. Statements and opinions expressed in the chapters are these of the individual contributors and not necessarily those of the editors or publisher. No responsibility is accepted for the accuracy of information contained in the published articles. The publisher assumes no responsibility for any damage or injury to persons or property arising out of the use of any materials, instructions, methods or ideas contained in the book. Publishing Process Manager Romina Krebel Technical Editor Teodora Smiljanic Cover Designer Jan Hyrat Image Copyright Kesu, 2010. Used under license from Shutterstock.com First published July, 2011 Printed in Croatia A free online edition of this book is available at www.intechopen.com Additional hard copies can be obtained from orders@intechweb.org Gene Therapy Applications, Edited by Chunsheng Kang p. cm. ISBN 978-953-307-541-9 free online editions of InTech Books and Journals can be found at www.intechopen.com Contents Preface IX Part 1 Gene Therapy in Blood and Vascular System 1 Chapter 1 Gene Therapy with Non-Viral Vectors For Critical Limb Ischemia: From Bench to Bedside 3 Erich Vinicius De Paula Chapter 2 Cell-Based Gene Therapies and Stem Cells for Regeneration of Ischemic Tissues 17 Rosalinda Madonna and Raffaele De Caterina Chapter 3 Establishment of Conditional Transgenic Mice Model with Cavernous Hemangioma Using the Tet-On System 35 Jia Wei Zheng Chapter 4 Potential Gene Therapy: Intravenous Administration of Phagocytes Transfected Ex Vivo with FGF4 DNA/Biodegradable Gelatin Complex Promotes Angiogenesis in Animal Model of Myocardial Ischemia/Reperfusion Injury 45 Toru Shizuma, Chiharu Tanaka, Hidezo Mori and Naoto Fukuyam Chapter 5 Approaches in Gene Therapy of Cancer and Cardiovascular Diseases 59 Gardlik Roman, Dovinova Ima and Chan Julie Y.H. Chapter 6 Rescue of Familial Hypertrophic Cardiomyopathy by Altering Sarcomeric Exposure and Response to Calcium 85 David F. Wieczorek and Beata M. Wolska Chapter 7 Gene Therapy in Cardiovascular Disease 95 José Luis Reyes-Juárez and Angel Zarain-Herzberg VI Contents Part 2 Gene Therapy in Orthopedics 127 Chapter 8 Potential Gene Therapy for Intervertebral Disc Degeneration 129 Kotaro Nishida, Koichiro Maeno, Kakutani Kenichiro, Takashi Yurube, and Masahiro Kurosaka Chapter 9 Conditioning and Scaffolding of Chondrocytes: Smart Steps Towards Osteoarthritis Gene Therapy 137 Muhammad Farooq Rai, Annemarie Lang, Matthias Sieber and Michael F.G. Schmidt Chapter 10 Gene Therapy Challenges in Arthritis 165 Denys Anne, Thiolat Allan, Boissier Marie-Christophe and Bessis Natacha Chapter 11 Gene Therapy Outcomes in Experimental Models of Inflammatory Arthritis 189 Charles J. Malemud Chapter 12 Gene Therapy Applications for Fracture Repair 201 Cassandra A. Strohbach, Donna D. Strong and Charles H. Rundle Chapter 13 Ex Vivo Gene Therapy for Spinal Fusion 227 Takashi Kaito, Stephanie S. Ngo and Jeffrey C. Wang Part 3 Gene Therapy in Genitourinary System 241 Chapter 14 Gene Therapy in Urology 243 Ratha Mahendran, Sin Mun Tham and Kesavan Esuvaranathan Chapter 15 Delivery Methods to Target RNAs in the Kidney 263 Csaba Révész and Péter Hamar Chapter 16 Therapeutic Potential of Gene Transfer to Testis; Myth or Reality? 279 Yoshiyuki Kojima, Kentaro Mizuno, Yukihiro Umemoto, Shoichi Sasaki, Yutaro Hayashi and Kenjiro Kohri Chapter 17 Quality of Life of Patients with Hormone Refractory Prostate Cancer After Gene Therapy 297 Akinobu Gotoh, Shuji Terao and Toshiro Shirakawa Chapter 18 Fetal Gene Therapy 307 Christopher Porada and Graça Almeida-Porada Contents VII Part 4 Gene Therapy in Other Diseases 325 Chapter 19 Genetic Addiction Risk Score (GARS): Testing For Polygenetic Predisposition and Risk to Reward Deficiency Syndrome (RDS) 327 Kenneth Blum, Frank Fornari, B.William Downs, Roger L. Waite, John Giordano, Andrew Smolen, Yijun Lui, Jai Tain, Neil Majmundar and Eric R. Braverman Chapter 20 Current and Future Biological Treatments in Inflammatory Bowel Disease 363 Jesus K. Yamamoto-Furusho Chapter 21 Gene Therapy for Alpha-1-Antitrypsin Deficiency Diseases 375 Parveen Salahuddin Chapter 22 Recent Developments in Gene Therapy Research Targeted to Cerebellar Disorders 401 Hirokazu Hirai and Akira Iizuka Chapter 23 Role of Gene Therapy in the Management of Unilateral Vocal Fold Paralysis 423 Kevin Fung Chapter 24 Fluorescence Cross-Correlation Spectroscopy for Real-Time Monitoring of Exogenous DNA Behavior in Living Cells 429 Akira Sasaki and Masataka Kinjo Chapter 25 Alpha-1 Antitrypsin Deficiency: Recent Developments in Gene Therapy Research 449 Catherine M. Greene and Noel G. McElvaney Chapter 26 Critical Stages in the Development of the First Targeted, Injectable Molecular-Genetic Medicine for Cancer 461 Erlinda M. Gordon and Frederick L. Hall Preface In the face of extraordinary advances in the prevention, diagnosis, and treatment of human diseases, devastating illnesses such as heart disease, and cancer continue to deprive people of health. Recently, techniques have been developed for gene therapy, providing unprecedented opportunities for further studying and understanding hu- man diseases. Although it is impossible to cure all human diseases, scientists and the public will gain immense new knowledge in the development of gene therapy that will likely hold remarkable potential for therapies. This book aims to cover key aspects of the potential and existing problems in the emerging field of gene therapy application. With the contribution of leading pioneers in various disciplines of gene therapy, the book brings together major approaches of gene therapy application in one text. Given that a great deal of data has been gathered and insights have been provided by researchers around the world, we believe that it will provide detailed clinical experiences and facilitate research in gene therapy. Dr. Chunsheng Kang Professor at Lab of Neuro-oncology Tianjin Neurological Institute China [...]... activation of other genes, the over-expression of which may cause cancer, as experienced when using ex vivo gene therapy for the treatment of children with Xlinked severe combined immune deficiency (Gansbacher & European Society of Gene 26 Gene Therapy Applications Therapy, 2003) Progress in the field of gene therapy has been limited by safety concerns related to delivery vectors Genetically modified... several characteristics render CLI a very interesting target for gene therapy- based therapeutic angiogenesis In this chapter we reviewed the molecular and cellular rationale of therapeutic angiogenesis, results from pre-clinical studies, and finally results from clinical trials that used gene therapy as a platform for therapeutic angiogenesis In addition, we tried to provide a critical appraisal of... associated with increased angiogenesis, such as inflammation, proliferative retinopathy and growth and spread of tumors (Carmeliet, 2003) In general terms, it is acknowledged that post-natal angiogenesis recapitulates the mechanisms described in the embryo 2.3 Gene therapy as the preferred platform for therapeutic angiogenesis One of the most attractive aspects of therapeutic angiogenesis is that by increasing... 1 Gene Therapy in Blood and Vascular System 1 Gene Therapy with Non-Viral Vectors For Critical Limb Ischemia: From Bench to Bedside Erich Vinicius De Paula Hematology and Hemotherapy Center, University of Campinas, Campinas, SP Brazil 1 Introduction As knowledge about the cellular and molecular mechanisms that control vessel growth grew during the last two decades, therapeutic manipulation of angiogenesis... survival in the injured myocardium In general, because of 18 Gene Therapy Applications the short half-lives of GFs in the body and the necessity to deliver them to specific target sites, GF injections themselves do not always produce the anticipated therapeutic effect At present, GF delivery in regenerative medicine basically relies upon two strategies: 1) delivery of the GF genes; 2) direct delivery of GFs... patient, transferring the gene( s) to the cells in tissue culture and then transferring the genetically modified cells back into the patient (ex vivo) In another recent review article we have specifically discussed cell-mediated HGF/Met gene transfer for myocardial regeneration (Madonna et al., 2010) A more general overview of preclinical studies using in vivo and ex vivo gene delivery strategies is... level of expectation that was probably too unrealistic considering the amount of basic and translational research on post-natal angiogenesis available at that time Gene transfer was rapidly considered the preferred method for therapeutic angiogenesis Using gene therapy (GT), genes of vascular growth factors could be directly injected into ischemic muscle, which would function both as the major therapeutic... claudication in subjects with peripheral arterial disease Am Heart J, Vol.153, No.5,(May), pp.874880 Gupta, R., J Tongers, et al (2009) Human studies of angiogenic gene therapy Circ Res, Vol.105, No.8,(Oct 9), pp.724-736 14 Gene Therapy Applications Habib, G B., J Heibig, et al (1991) Influence of coronary collateral vessels on myocardial infarct size in humans Results of phase I thrombolysis in myocardial... endpoints occurred in both treated and control groups, highlighting the importance of placebo effect in studies using new strategies such as gene therapy 4.2 Safety concerns To date, more than 1000 subjects have been treated for gene therapy for therapeutic angiogenesis in phase I and II trials, with adverse event rates that are similar to those in control groups (Varu et al., 2010) Still, there are... Menasche et al., 2003; Pagani et al., 2003; Smits et al., 2003) Bone marrow-derived stem cells are currently the most commonly used cells in cell transplantation therapy The ideal stem 20 Gene Therapy Applications cells from bone marrow for cardiac regeneration remain to be identified and many details remain to be elucidated Yet, the clinical results from recent trials show the capability of these cells . GENE THERAPY APPLICATIONS Edited by Chunsheng Kang Gene Therapy Applications Edited by Chunsheng Kang Published. Chapter 7 Gene Therapy in Cardiovascular Disease 95 José Luis Reyes-Juárez and Angel Zarain-Herzberg VI Contents Part 2 Gene Therapy in Orthopedics 127 Chapter 8 Potential Gene Therapy for. Chapter 13 Ex Vivo Gene Therapy for Spinal Fusion 227 Takashi Kaito, Stephanie S. Ngo and Jeffrey C. Wang Part 3 Gene Therapy in Genitourinary System 241 Chapter 14 Gene Therapy in Urology