Figure 1 summarises the main steps necessary to push forward an AAV-based gene therapy medicinal product from preclinical studies to clinical trials Without minimising the importance of the tragic toxic events seen in the current clinical trials, it is worth emphasizing that AAV-mediated gene therapy is the only treatment that led to highly significant disease improvement in severely affected human patients.
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Of rAAV and Men: From Genetic Neuromuscular Disorder Efficacy and Toxicity Preclinical Studies to Clinical Trials and Back.
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