What Is Cell And Gene Therapy?

For patients whose treatment has failed or doesn't exist yet, cell and gene therapy tries something different: fix the fault at its source, ideally for good. Children born with a fatal single-gene condition can receive a working copy of the gene. In some cases their own cells are removed, engineered with the new gene and returned; in others the gene is delivered directly to the tissue that needs it. In cancer, immune cells are upgraded to recognize rogue cells and kill them, the approach known as CAR T. A permanent cure in place of a lifetime of treatment would change what medicine can promise. A short overview also looks at where development and manufacturing come in. Read it for the basics and the role of the people who make these therapies.