Gene Transfer, Genome Editing and Gene Therapy: Principles and Strategies

Gene transfer, genome editing, and gene therapy are recent technologies which have revolutionized the traditional concepts of therapeutic pharmacology. Over the last 10 years they have led to spectacular and groundbreaking therapeutic and vaccine successes, transformed biotechnology, the life sciences, and medicine by treating previously uncurable diseases, and become critical to biology laboratories all over the world.This basic and interdisciplinary textbook describes the essential concepts and techniques which have been developed in gene therapy and gene manipulation. The difference between in vivo and ex vivo gene delivery strategies is explored. The principles of viral and non-viral chemical and physical gene delivery techniques are presented. The science behind and modern use of antisense oligonucleotides, exon skipping, mRNA, and small interfering RNAs are explained, with specific chapters dedicated to describing 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 post-graduate students, researchers, physicians, and industry scientists -- will find this text to be essential reading, supported by the included presentation slides and quiz, which foster a developed and comprehensive understanding of the many different topics.