Open Access Medical Books



Edited by Francisco Martin Molina .

745 pages . 

In the last 10 years gene therapy has experienced a renascence thanks to the development of safer and more efficient gene transfer vectors and to the advances in the cell therapy field.
This book brings together a comprehensive collection of gene therapy tools and their therapeutic applications. The first part of the book covers different gene therapy vectors focusing on their advantages and disadvantages. The second part of the book gets into gene therapy applications, from the latest successes on clinical trials to the new gene therapy targets that are still under development. This book allows the reader to come across with the opinions of different experts in the gene therapy field.

Francisco Martín Molina
Principal Investigator
Gene and Cell Therapy group
Pfizer - Universidad de Granada - Junta de Andalucía
 Centre for Genomics and
Oncological Research (GENYO)


Section 1 : Introduction .

 1 Non-Viral Delivery Systems in Gene Therapy 3
Alicia Rodríguez Gascón, Ana del Pozo-Rodríguez and María Ángeles Solinís

 2 Plasmid Transgene Expression in vivo: Promoter and Tissue Variables 35
David Morrissey, Sara A. Collins, Simon Rajenderan, Garrett Casey, Gerald C. O’Sullivan and Mark Tangney

 3 Silencing of Transgene Expression: A Gene Therapy Perspective 49
Oleg E. Tolmachov, Tatiana Subkhankulova and Tanya Tolmachova

Section 2 : Gene Therapy Tools: Synthetic .

 4 Cellular Uptake Mechanism of Non-Viral Gene Delivery and Means for Improving Transfection Efficiency 71
Shengnan Xiang and Xiaoling Zhang

 5 Polylipid Nanoparticle, a Novel Lipid-Based Vector for Liver Gene Transfer 91
Yahan Fan and Jian Wu

 6 DNA Electrotransfer: An Effective Tool for Gene Therapy 109
Aurore Burgain-Chain and Daniel Scherman

 7 siRNA and Gene Formulation for Efficient Gene Therapy 135
Ian S. Blagbrough and Abdelkader A. Metwally

Section 3 : Gene Therapy Tools: Biological .

 8 Mesenchymal Stem Cells as Gene Delivery Vehicles 177
Christopher Porada and Graa Almeida-Porada

 9 Cancer Gene Therapy – Key Biological Concepts in the Design of Multifunctional Non-Viral Delivery Systems 213
Cian M. McCrudden and Helen O. McCarthy

 10 Gene Therapy Based on Fragment C of Tetanus Toxin in ALS: A Promising Neuroprotective Strategy for the Bench to the Bedside Approach 249
Ana C. Calvo, Pilar Zaragoza and Rosario Osta

 11 Transposons for Non-Viral Gene Transfer 269
Sunandan Saha and Matthew H. Wilson

 12 Lentiviral Gene Therapy Vectors: Challenges and Future Directions 287
Hélio A. Tomás, Ana F. Rodrigues, Paula M. Alves and Ana S. Coroadinha

 13 Lentiviral Vectors in Immunotherapy 319
Ines Dufait, Therese Liechtenstein, Alessio Lanna, Roberta Laranga, Antonella Padella, Christopher Bricogne, Frederick Arce, Grazyna Kochan, Karine Breckpot and David Escors

 14 Targeted Lentiviral Vectors: Current Applications and Future Potential 343
Cleo Goyvaerts, Therese Liechtenstein, Christopher Bricogne, David Escors and Karine Breckpot

 15 Vectors for Highly Efficient and Neuron-Specific Retrograde Gene Transfer for Gene Therapy of Neurological Diseases 387
Shigeki Kato, Kenta Kobayashi, Ken-ichi Inoue, Masahiko Takada and Kazuto Kobayashi

 16 Retroviral Genotoxicity 399
Dustin T. Rae and Grant D. Trobridge

 17 Efficient AAV Vector Production System: Towards Gene Therapy For Duchenne Muscular Dystrophy 429
Takashi Okada

Section 4 : Applications: Inhereted Diseases .

 18 Gene Therapy for Primary Immunodeficiencies 453
Francisco Martin, Alejandra Gutierrez-Guerrero and Karim Benabdellah

 19 Gene Therapy for Diabetic Retinopathy – Targeting the Renin-Angiotensin System 467
Qiuhong Li, Amrisha Verma, Ping Zhu, Bo Lei, Yiguo Qiu, Takahiko Nakagawa, Mohan K Raizada and William W Hauswirth

 20 Gene Therapy for Retinitis Pigmentosa 493
Hiroshi Tomita, Eriko Sugano, Hitomi Isago, Namie Murayama and Makoto Tamai

 21 Gene Therapy for Erythroid Metabolic Inherited Diseases 511
Maria Garcia-Gomez, Oscar Quintana-Bustamante, Maria Garcia- Bravo, S. Navarro, Zita Garate and Jose C. Segovia

 22 Targeting the Lung: Challenges in Gene Therapy for Cystic Fibrosis 539
George Kotzamanis, Athanassios Kotsinas, Apostolos Papalois and Vassilis G. Gorgoulis

 23 Gene Therapy for the COL7A1 Gene 561
E. Mayr, U. Koller and J.W. Bauer

 24 Molecular Therapy for Lysosomal Storage Diseases 591
Daisuke Tsuji and Kohji Itoh

Section 5 : Applications: Others .

 25 Gene Therapy Perspectives Against Diseases of the Respiratory System 611
Dimosthenis Lykouras, Kiriakos Karkoulias, Christos Tourmousoglou, Efstratios Koletsis, Kostas Spiropoulos and Dimitrios Dougenis

 26 Gene Therapy in Critical Care Medicine 631
Gabriel J. Moreno-González and Angel Zarain-Herzberg

 27 Clinical and Translational Challenges in Gene Therapy of Cardiovascular Diseases 651
Divya Pankajakshan and Devendra K. Agrawal

 28 Gene Therapy for Chronic Pain Management 685
Isaura Tavares and Isabel Martins

 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 703
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

 30 Feasibility of Gene Therapy for Tooth Regeneration by Stimulation of a Third Dentition 727
Katsu Takahashi, Honoka Kiso, Kazuyuki Saito, Yumiko Togo, Hiroko Tsukamoto, Boyen Huang and Kazuhisa Bessho .

You've just read an article category Biochemistry Genetics and Molecular Biology by title GENE THERAPY - TOOLS AND POTENTIAL APPLICATIONS. You can bookmark this page URL Thank you!
Published by: younes younes - Thursday, February 28, 2013


  1. It is very good blog.I am also looking for this from a long time.Many people like to visit here.I have seen many things over here.It is very good.

    Ophthalmic Biometry