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[The development of a therapy for human use entails a complex and long process of studies and validations, first in cells and animal models and later in human subjects. The first step is commonly referred to as preclinical research or development, being important to access the safety and also the efficacy of the therapy proposed. In a gene therapy context, the in vivo preclinical studies, using relevant animal models, are particularly important, as most of the therapies are being studied for the first time. If preclinical studies yield relevant results, therapy can continue its process of development, being then evaluated in clinical trials, which refer to research studies performed in human subjects. In their simplest design, clinical trials aim to evaluate the outcomes of a particular therapy in human subjects under an experimental condition, being also referred to as interventional clinical studies.]
Published: Jun 28, 2020
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