Gene Therapy
摘要
Gene therapy is a relatively new medical modality that involves using genetic material or modifying the genetic material of a cell as a therapeutic tool. This chapter will first describe the characteristics of the therapeutic genetic material and the main vectors used for their delivery to the cell. Following this, it will describe the different types of gene therapy modalities according to the mode of administration and mechanism of action, with special attention to new therapeutic strategies based on gene editing. The chapter will conclude by describing the gene therapy products that have reached the market and the limitations encountered in their global distribution.