Open access peer-reviewed Edited Volume

In Vivo and Ex Vivo Gene Therapy for Inherited and Non-Inherited Disorders

Ongoing advances in pharmaceutical biotechnology have paved the way to ground-breaking new biological therapeutic modalities, offering the possibility of a durable curative approach for a number of life-threatening diseases, for which the medical need is as yet unmet. Over the past decades, gene therapy has seen a massive transformation from a proof-of-concept approach to a clinical reality culminating in the regulatory approval of state-of-the-art products in the European Union and in the United States. This book captures some of the scientific progresses notably in gene transfer technologies and translational development of in vivo and ex vivo gene therapy interventions in the treatment of a broad range of complex and debilitating non-inherited and inherited disorders such as: human immunodeficiency virus 1 (HIV-1) infection, cancer, cystic fibrosis, hereditary retinopathies, haemophilia B, cardiac diseases, and chronic liver fibrosis.

Read more >Order hardcopy
In Vivo and Ex Vivo Gene Therapy for Inherited and Non-Inherited DisordersEdited by Houria Bachtarzi

Published: March 13th 2019

DOI: 10.5772/intechopen.72962

ISBN: 978-1-78985-718-4

Print ISBN: 978-1-78985-717-7

eBook (PDF) ISBN: 978-1-83962-082-9

Copyright year: 2019

Books open for chapter submissions

8921 Total Chapter Downloads

4 Crossref Citations

1 Web of Science Citations

7 Dimensions Citations


Open access peer-reviewed

1. Nucleic Acid-Based Therapy: Development of a Nonviral-Based Delivery Approach

By Takeshi Yokoo, Kenya Kamimura, Tsutomu Kanefuji, Takeshi Suda and Shuji Terai


Open access peer-reviewed

2. Gene Therapy for Cystic Fibrosis: Hurdles to Overcome for Successful Clinical Translation

By Myriam Sainz-Ramos, Nuseibah AL Qtaish, Idoia Gallego, Ilia Villate- Beitia, Tania López, Gustavo Puras and José Luis Pedraz


Open access peer-reviewed

3. Mechanisms for Controlling HIV-1 Infection: A Gene Therapy Approach

By Katherine Ognenovska, Vera Klemm, Scott Ledger, Stuart Turville, Geoff Symonds, Anthony D. Kelleher and Chantelle L. Ahlenstiel


Open access peer-reviewed

4. Functional Activation of Autologous Human Diabetic Stem Cells for Cell Therapy

By Patrick L. Iversen, Francis W. Ruscetti, Charles Garcia and Stephen H. Bartelmez


Open access peer-reviewed

5. Gene-based Interventions for Cancer Immunotherapy

By Otto L.D. Cerqueira, Gissele Rolemberg Oliveira Silva, Igor de Luna Vieira, Marlous Vinícius Gomes Lana, Nadine Gimenez, Otavio Augusto Rodrigues, Paulo Roberto Del Valle, Samir Andrade Mendonça and Bryan E. Strauss


Open access peer-reviewed

6. AAV-Mediated Gene Therapy for CRB1-Hereditary Retinopathies

By Celso Henrique Alves and Jan Wijnholds


Open access peer-reviewed

7. Adeno-Associated Virus (AAV)-Mediated Gene Therapy for Disorders of Inherited and Non-Inherited Origin

By Indu Rajapaksha, Peter Angus and Chandana Herath


Open access peer-reviewed

8. Gene Therapy for Cardiomyopathies

By Yves Fromes and Caroline Roques


Edited Volume and chapters are indexed in

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

Order a hardcopy of the Edited Volume

Free shipping with DHL Express

Hardcover (ex. VAT)£119

Order now

Residents of European Union countries need to add a Book Value-Added Tax Rate based on their country of residence. Institutions and companies, registered as VAT taxable entities in their own EU member state, will not pay VAT by providing IntechOpen with their VAT registration number. This is made possible by the EU reverse charge method.

Special discount for IntechOpen contributors

All IntechOpen contributors are offered special discounts starting at 40% OFF available through your personal dashboard

Login and purchase