Human Stem Cell Gene Therapy

Human embryonic and adult stem cells hold great promise for the treatment of a wide variety of acquired and inherited human diseases including congestive heart failure, neurodegenerative diseases (e.g. Parkinson’s disease), liver cirrhosis, cerebral infarction, muscular dystrophy and autoimmune diseases (e.g. type I diabetes) [1–[4]]. Apart from their use to restore or replace tissues that have been damaged, worn out or lost as a result of disease, senescence or trauma, human stem cells (hSCs) may be excellent tools to study the genes and proteins controlling embryonic development, cell differentiation and oncogenic transformation. Other possible applications of hSCs and their derivatives include the testing of novel drugs, the screening of potential toxins and the production of therapeutic proteins. To exploit the full potential of hSCs it will be important to develop safe and efficient methods for the stable transduction of these cells.