Gene Transfer, Genome Editing and Gene Therapy

This basic and interdisciplinary textbook describes the essential concepts and techniques that have been developed for gene transfer, genome editing, and gene therapy. These are recent technologies that have revolutionized conventional approaches to therapy and pharmacology. Over the last 10 years, these technologies have led to spectacular and groundbreaking therapeutic and vaccine successes, transformed biotechnology, life sciences, and medicine by treating previously incurable diseases, and become critical to biology laboratories all over the world.

Topics such as the difference between in vivo and ex vivo gene delivery strategies, the principles of viral and non-viral chemical and physical gene delivery techniques, and the science behind modern use of antisense oligonucleotides, exon skipping, mRNA, and small interfering RNAs are fully explored and explained. However, the book also goes beyond these fundamentals to engage with advanced details such as anticancer personalized adoptive immunotherapy using CAR-T cells and CRISPR/Cas9 nucleoprotein complex use for genome editing.

Anyone interested in biotechnology and the recent genetic medicine revolution — including graduate, advanced undergraduate, and postgraduate students, researchers, physicians, pharmacologists, veterinarians, and industry scientists — will find this text to be essential reading. The content is supplemented with presentation slides and quiz questions, fostering a thorough and comprehensive understanding of the subject.

Daniel Scherman is Emeritus Research Director at the CNRS National Scientific Research Center and at Université Paris Cité. He has dedicated most of his scientific activity to the gene therapy field, both in terms of delivery methods and for therapeutic applications. He has served several terms in the past and presently on the Scientific Committee of the American Society of Gene and Cell Therapy and as the scientific director of the Généthon Laboratory dedicated to the gene therapy of rare genetic diseases. Professor Daniel Scherman also manages the French Foundation for Rare Diseases.

He is presently the head of the Life Sciences and Medicine Division of the European Academy of Sciences EURASC, and an associated member of the French Academy of Pharmacy.