Pioneering Stem Cell/Gene Therapy Trials

Inbunden, Engelska, 2003

AvRoger Bertolotti,Keiyo Ozawa,H. Kirk Hammond

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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.This volume will be of value and interest to researchers in this exciting field.

Produktinformation

Utgivningsdatum2003-10-15
Mått156 x 234 x undefined mm
Vikt776 g
FormatInbunden
SpråkEngelska
Antal sidor330
FörlagBrill
ISBN9789067643948

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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.

Introduction; 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