Development of gene therapy using genome editing technology

Abstract

 A current definition of gene therapy is the infusion of a therapeutic gene, or cells transduced with the gene, in order to cure or prevent illnesses. This method can be used to add a functional gene into genomes containing a mutated version, which is left unmodified. However, gene therapy cannot be used to treat diseases caused by gain-of-function mutations, and leukemogenesis is often induced by integration of the vector into sites adjacent to proto-oncogenes. On the other hand, gene editing technologies, such as the CRISPR/Cas system, have been developed at a rapid pace and introduced into the field of gene therapy. This article describes the safety, efficacy, and ethics of gene therapy, by focusing on problems inherent to current gene therapy techniques involving gene addition, and the development of future gene therapies using gene correction.