Principles of Therapeutics and Gene-Based Therapy

This chapter focuses on the history and evolution of gene-based therapies from its inception in the early 1970s to the landmark discovery of the CRISPR-Cas9 gene editing, which was ultimately recognized and awarded the Noble Prize in 2020. This chapter reviews essential requirements of traditional gene therapy approach and lists the gene-based therapies currently marketed in different countries, in conjunction with their associated limitations and concerns. This chapter also discusses in-depth CRISPR-Cas9 technology as an efficient alternative for site-specific gene alteration that practically eliminates many red flags raised by traditional gene therapy methods. The limitations associated with the CRISPR-Cas9 gene editing methods have been afforded their due attention within the scope of the discussion.