Clinical Safety and Applications of Stem Cell Gene Therapy

Gene therapy is an exciting new field that has the potential to contribute to the treatment of many incurable diseases. Ensuring safety and efficacy are key objectives. Here we review the latest developments in gene therapy vectors, the stem cell transduction techniques, and the therapeutic applications under investigation. The development of stem cell gene therapies is complex and requires optimization of many diverse elements. Local and international consortia will facilitate the entry of these technologies into the clinic through the sharing of knowledge and provision of guidance and support.