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.