**Meet the editor**

Dr Houria Bachtarzi is a Senior Consultant, Regulatory Affairs - Gene and Cell Therapies, with vast scientific and regulatory experience in the field of advanced biological therapies including virally vectored gene therapy, *in vivo* and *ex vivo* gene editing, cell-based immunotherapy, genetically modified cells for tissue regeneration, genetically engineered immune cells tar-

geting cancer cells, and stem cell-based therapies. She was previously a Lecturer in Biopharmaceutics and Biotechnology within the Pharmacology and Therapeutics Division at the School of Pharmacy and Biomolecular Sciences, University of Brighton, UK. Houria completed her PhD in cancer gene therapy/viral gene delivery at the University of Oxford, Department of Oncology, followed by Post-Doctoral research work in AAV-based gene therapy and shRNA therapeutics for degenerative neuro-muscular disorders at the Centre for Biomedical Sciences, Royal Holloway-University of London. Houria holds a First Class Master of Pharmacy Degree (with honors) from the University of Bath, UK and is a Registered Pharmacist with the General Pharmaceutical Council in the UK. She is also a Member of the Royal Pharmaceutical Society of Great Britain (RPSGB) and a Member of the British Society for Gene and Cell Therapy (BSGCT). Houria has published a number of peer-reviewed papers in gene therapy and has presented platform presentations at many international and national scientific meetings. In 2014, she received a Rising Star Award from the University of Brighton.

Contents

**Preface VII**

**Applications 1**

and Shuji Terai

**Approach 41**

Bartelmez

**Cells for Cell Therapy 69**

**and Other Genetic Diseases 87**

Mendonça and Bryan E. Strauss

**Delivery Approach 3**

**Section 1 Gene Transfer Technologies for In Vivo and Ex Vivo**

Chapter 1 **Nucleic Acid-Based Therapy: Development of a Nonviral-Based**

Chapter 2 **Gene Therapy for Cystic Fibrosis: Hurdles to Overcome for**

Beitia, Tania López, Gustavo Puras and José Luis Pedraz

Chapter 3 **Mechanisms for Controlling HIV-1 Infection: A Gene Therapy**

Chapter 4 **Functional Activation of Autologous Human Diabetic Stem**

**Section 2 Advances in Virotherapy: Virus-based Gene Therapy for Cancer**

Chapter 5 **Gene-based Interventions for Cancer Immunotherapy 89**

**Successful Clinical Translation 21**

Takeshi Yokoo, Kenya Kamimura, Tsutomu Kanefuji, Takeshi Suda

Myriam Sainz-Ramos, Nuseibah AL Qtaish, Idoia Gallego, Ilia Villate-

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

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

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
