Open access peer-reviewed Edited Volume

Gene Therapy

Tools and Potential Applications

The Gene Therapy field is living exciting times after more than 20 years of poor results. Scientist and clinicians working in the gene therapy field have encountered many problems in the past that are now starting to be solved. The development of safer and more efficient gene transfer vectors and the advances on the cell therapy field have open new opportunities to tackle different diseases. The aim of this book is to bring together information about the different gene therapy tools, the clinical successes of gene therapy and the future applications.

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Gene TherapyTools and Potential ApplicationsEdited by Francisco Martin

Published: February 27th 2013

DOI: 10.5772/50194

ISBN: 978-953-51-1014-9

eBook (PDF) ISBN: 978-953-51-5362-7

Copyright year: 2013

Books open for chapter submissions

67076 Total Chapter Downloads

41 Crossref Citations

105 Web of Science Citations

125 Dimensions Citations


Open access peer-reviewed

1. Non-Viral Delivery Systems in Gene Therapy

By Alicia Rodríguez Gascón, Ana del Pozo-Rodríguez and María Ángeles Solinís


Open access peer-reviewed

2. Plasmid Transgene Expression in vivo: Promoter and Tissue Variables

By David Morrissey, Sara A. Collins, Simon Rajenderan, Garrett Casey, Gerald C. O’Sullivan and Mark Tangney


Open access peer-reviewed

3. Silencing of Transgene Expression: A Gene Therapy Perspective

By Oleg E. Tolmachov, Tatiana Subkhankulova and Tanya Tolmachova


Open access peer-reviewed

4. Cellular Uptake Mechanism of Non-Viral Gene Delivery and Means for Improving Transfection Efficiency

By Shengnan Xiang and Xiaoling Zhang


Open access peer-reviewed

5. Polylipid Nanoparticle, a Novel Lipid-Based Vector for Liver Gene Transfer

By Yahan Fan and Jian Wu


Open access peer-reviewed

6. DNA Electrotransfer: An Effective Tool for Gene Therapy

By Aurore Burgain-Chain and Daniel Scherman


Open access peer-reviewed

7. siRNA and Gene Formulation for Efficient Gene Therapy

By Ian S. Blagbrough and Abdelkader A. Metwally


Open access peer-reviewed

8. Mesenchymal Stem Cells as Gene Delivery Vehicles

By Christopher Porada and Graça Almeida-Porada


Open access peer-reviewed

9. Cancer Gene Therapy – Key Biological Concepts in the Design of Multifunctional Non-Viral Delivery Systems

By Cian M. McCrudden and Helen O. McCarthy


Open access peer-reviewed

10. Gene Therapy Based on Fragment C of Tetanus Toxin in ALS: A Promising Neuroprotective Strategy for the Bench to the Bedside Approach

By Ana C. Calvo, Pilar Zaragoza and Rosario Osta


Open access peer-reviewed

11. Transposons for Non-Viral Gene Transfer

By Sunandan Saha and Matthew H. Wilson


Open access peer-reviewed

12. Lentiviral Gene Therapy Vectors: Challenges and Future Directions

By Hélio A. Tomás, Ana F. Rodrigues, Paula M. Alves and Ana S. Coroadinha


Open access peer-reviewed

13. Lentiviral Vectors in Immunotherapy

By Ines Dufait, Therese Liechtenstein, Alessio Lanna, Roberta Laranga, Antonella Padella, Christopher Bricogne, Frederick Arce, Grazyna Kochan, Karine Breckpot and David Escors


Open access peer-reviewed

14. Targeted Lentiviral Vectors: Current Applications and Future Potential

By Cleo Goyvaerts, Therese Liechtenstein, Christopher Bricogne, David Escors and Karine Breckpot


Open access peer-reviewed

15. Vectors for Highly Efficient and Neuron-Specific Retrograde Gene Transfer for Gene Therapy of Neurological Diseases

By Shigeki Kato, Kenta Kobayashi, Ken-ichi Inoue, Masahiko Takada and Kazuto Kobayashi


Open access peer-reviewed

16. Retroviral Genotoxicity

By Dustin T. Rae and Grant D. Trobridge


Open access peer-reviewed

17. Efficient AAV Vector Production System: Towards Gene Therapy For Duchenne Muscular Dystrophy

By Takashi Okada


Open access peer-reviewed

18. Gene Therapy for Primary Immunodeficiencies

By Francisco Martin, Alejandra Gutierrez-Guerrero and Karim Benabdellah


Open access peer-reviewed

19. Gene Therapy for Diabetic Retinopathy – Targeting the Renin-Angiotensin System

By Qiuhong Li, Amrisha Verma, Ping Zhu, Bo Lei, Yiguo Qiu, Takahiko Nakagawa, Mohan K Raizada and William W Hauswirth


Open access peer-reviewed

20. Gene Therapy for Retinitis Pigmentosa

By Hiroshi Tomita, Eriko Sugano, Hitomi Isago, Namie Murayama and Makoto Tamai


Open access peer-reviewed

21. Gene Therapy for Erythroid Metabolic Inherited Diseases

By Maria Garcia-Gomez, Oscar Quintana-Bustamante, Maria Garcia- Bravo, S. Navarro, Zita Garate and Jose C. Segovia


Open access peer-reviewed

22. Targeting the Lung: Challenges in Gene Therapy for Cystic Fibrosis

By George Kotzamanis, Athanassios Kotsinas, Apostolos Papalois and Vassilis G. Gorgoulis


Open access peer-reviewed

23. Gene Therapy for the COL7A1 Gene

By E. Mayr, U. Koller and J.W. Bauer


Open access peer-reviewed

24. Molecular Therapy for Lysosomal Storage Diseases

By Daisuke Tsuji and Kohji Itoh


Open access peer-reviewed

25. Gene Therapy Perspectives Against Diseases of the Respiratory System

By Dimosthenis Lykouras, Kiriakos Karkoulias, Christos Tourmousoglou, Efstratios Koletsis, Kostas Spiropoulos and Dimitrios Dougenis


Open access peer-reviewed

26. Gene Therapy in Critical Care Medicine

By Gabriel J. Moreno-González and Angel Zarain-Herzberg


Open access peer-reviewed

27. Clinical and Translational Challenges in Gene Therapy of Cardiovascular Diseases

By Divya Pankajakshan and Devendra K. Agrawal


Open access peer-reviewed

28. Gene Therapy for Chronic Pain Management

By Isaura Tavares and Isabel Martins


Open access peer-reviewed

29. Insulin Trafficking in a Glucose Responsive Engineered Human Liver Cell Line is Regulated by the Interaction of ATP-Sensitive Potassium Channels and Voltage-Gated Calcium Channels

By Ann M. Simpson, M. Anne Swan, Guo Jun Liu, Chang Tao, Bronwyn A O’Brien, Edwin Ch’ng, Leticia M. Castro, Julia Ting, Zehra Elgundi, Tony An, Mark Lutherborrow, Fraser Torpy, Donald K. Martin, Bernard E. Tuch and Graham M. Nicholson


Open access peer-reviewed

30. Feasibility of Gene Therapy for Tooth Regeneration by Stimulation of a Third Dentition

By Katsu Takahashi, Honoka Kiso, Kazuyuki Saito, Yumiko Togo, Hiroko Tsukamoto, Boyen Huang and Kazuhisa Bessho


Edited Volume and chapters are indexed in

  • Worldcat
  • OpenAIRE
  • Google Scholar
  • AZ ebsco
  • Base
  • CNKI

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