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.

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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

Copyright year: 2019

Books open for chapter submissions

1814 Total Chapter Downloads

1 Dimensions Citations

chaptersDownloads

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

208

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

195

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

256

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

181

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

184

Open access peer-reviewed

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

By Celso Henrique Alves and Jan Wijnholds

255

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

410

Open access peer-reviewed

8. Gene Therapy for Cardiomyopathies

By Yves Fromes and Caroline Roques

125

Edited Volume and chapters are indexed in

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

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