Description
Book SynopsisThe 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.
This volume will be of value and interest to researchers in this exciting field.
Table of ContentsIntroduction; Stem cell-gene therapy: a breakthrough combination magnified by therapeutic stem cell homing; Inherited disease: stem cells and life-long therapeutic gene expression paradigm; Clinical gene therapy trials for ADA deficiency and other Immunodeficiencies; Regenerative cardiovascular medicine: transient gene therapy and autologous stem cell transplantation 1. Transient angiogenic/vasculogenic gene therapy clinical trials Vascular endothelial growth factor (VEGF) gene therapy for critical limb ischemia 2. Autologous stem cell therapy: clinical trials for cardiovascular Disease 3. Synergyzing stem cell* and gene-therapy for cardiovascular Regeneration; Neurodegenerative disease: long-term gene therapy and neural/stem cell targets; Non-invasive monitoring of clinical gene therapy trials
