Gene Therapy

by Mauro Giacca

The overall purpose of this book is to expose the reader/student to a comprehensive and detailed, yet still easily accessible, overview of the whole field of gene therapy, ranging from vector development and therapeutic gene selection to the results of the most recent clinical trials. The language is plain and, whenever possible, non-technical. Since the book is intended to be a textbook in the field of gene therapy in both the basic science and clinical areas, when technical descriptions are required, they are provided either in the main text or in add-on explanatory boxes. For example, clinical readers might find it difficult to understand the principles of viral vector design without knowing some molecular details of viral genome organization and virus life cycle, and basic scientists might be unaware of the clinical and therapeutic advancements for the various disorders treated by the book. In these cases, basic explanations are provided. In this respect, it is worth pointing out that the book is truly a gene "therapy" book; it only considers conditions and experimental approaches based on gene transfer that have already proceeded to the clinic or are close to doing so. Basic research on gene transfer or nucleic acid modification is only presented if it is when close to clinical application. Following the same principle, only diseases for which clinical trials have already been performed or are planned are considered, or those for which solid and encouraging pre-clinical, animal experimentation results are available.

324 pages, Hardcover

First published April 22, 2010

About the author

Director, ICGEB Trieste Component

International Centre for Genetic Engineering and Biotechnology
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Education

Faculty of Medicine, University of Trieste, Italy, MD, 1984
Admitted to the Medical Doctors and Surgeons Register, 1985
University of Genova, Italy, PhD, 1989
Career History

Since July 2004, Director of the Trieste Component of the International Centre for Genetic Engineering and Biotechnology (ICGEB), Trieste, Italy.
2000-2004, Director of the Molecular Biology Laboratory of the Scuola Normale Superiore in Pisa, Italy.
Since 1995, Senior Scientist and Group Leader of the Molecular Medicine Laboratory of the ICGEB in Trieste.
1988-1994, Assistant Research Scientist at ICGEB, Trieste.
1986-1987, PhD student at the IGBE, CNR, Pavia.
1980-1985, Pre-doctoral student and fellow at the Institute of Microbiology of the University of Trieste.
Teaching Activity

Since 2005, Full Professor, University of Trieste.
2000-2005, Associate Professor of Molecular Biology at the Scuola Normale Superiore in Pisa.
Since 2001, external Professor in the course Gene Therapy at the Medical School at the University of Trieste.
1989-1999, external Professor in different courses of Microbiology and Medical Genetics at the Faculty of Medicine of the University of Trieste.
1991-1996, Lecturer in the PhD Course of Molecular Genetics of the International School for Advanced Studies (ISAS) of Trieste, Italy.
Scientific Activity

Current research interest focuses on the development of novel biotherapeutics for cardiovascular disorders, with special emphasis on the identification of growth factors and microRNAs able to induce new blood vessel formation and cardiac regeneration in patients with myocardial infarction or heart failure. He also maintains a strong interest in the molecular biology of HIV-1 infection.
Research activity is funded through grants from various public and private bodies, including the European Research Council (ERC Advanced Investigator Grants), the Italian Ministry for Education and Research, the World Antidoping Agency (WADA) Canada, the JDRF USA, the Telethon Foundation Italy, among others.
M. Giacca regularly reviews scientific manuscripts for international journals in the fields of gene therapy, cardiovascular disorders and HIV infection, and grant applications in the context of calls from the European Commission and various granting bodies throughout Europe and has published over 240 papers in peer-reviewed international journals and 20 reviews or chapters in published books and university text books. He is the author of the book "Gene Therapy", Springer ed., 2010. He regularly presents his research activity at meetings and seminars worldwide.