23/10/2022
Gene therapy strategies vary in invasiveness‼
In ex vivo gene therapy (“outside the body”), cells are altered outside the body and then placed in the body, such as through an intravenous infusion or into the spinal fluid. In the more invasive in vivo gene therapy (“in the living body”), the gene and its vector are introduced directly into the body, such as through a catheter inserted and snaked to the appropriate organ. There, the vector must enter the targeted cells, the human DNA be transcribed into mRNA, the mRNA translated into protein, and then the protein must function sufficiently to halt progression of the disease or improve symptoms.
Gene therapy invasiveness. Therapeutic genes are delivered to cells removed from the body that are then returned (ex vivo gene therapy) or delivered directly to an interior body part (in vivo gene therapy).
