Pioneering Stem Cell/Gene Therapy Trials

The first unequivocal success for Gene Therapy was reported in April 2000 for X-SCID patients. Pioneering stem cell/gene therapy clinical trials are the focus of this book. Therapy successes such as the X-SCID trial and improved ADA-SCID ones are presented together with pioneering angio/vasculogenic clinical trials mediated either by transient gene therapy or emerging autologous stem cell transplantation. Highlights also include 1) promises of the breakthrough combination of stem cell- and transient gene-therapy, 2) gene therapy trials for neurodegenerative disease on non-human primates where long-term gene therapy might involve brain stem cells, and 3) the first clinical trial with non-invasive monitoring of therapeutic gene expression as a prospective conclusion.

About the Author

Roger Bertolotti, CNRS, Molecular Genetics, Department of Hepato-Gastroenterology, Faculty of Medicine, University of Nice-Sophia Antipolis, France. Keiyo Ozawa, Division of Hematology, Department of Medicine, and Division of Cell Transplantation and Transfusion, and Division of Genetic Therapeutics, Center for Molecular Medicine, Jichi Medical School, Japan. H. Kirk Hammond, Division of Cardiology, VA San Diego Healthcare System and University of California, San Diego School of Medicine, San Diego, California.