Introduction to Gene Therapy: A Clinical Aftermath
Denèfle, Patrice P
Methods in molecular biology (Clifton, N.J.) 2011; 737: 27-44
Despite three decades of huge progress in molecular genetics, in cloning of disease causative gene as well as technology breakthroughs in viral biotechnology, out of thousands of gene therapy clinical trials that have been initiated, only very few are now reaching regulatory approval. We shall review some of the major hurdles, and based on the current either positive or negative examples, we try to initiate drawing a learning curve from experience and possibly identify the major drivers for future successful achievement of human gene therapy trials.
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French gene therapy group reports on the adverse event in a clinical trial of gene therapy for X-linked severe combined immune deficiency (X-SCID). Position statement from the European Society of Gene Therapy European Society of Gene Therapy [ESGT] (2003-01)