In the field of rare diseases the development of innovative technologies to study potential benefits of targeted medications and the analysis of effects of a potential gene therapy is substantial. The goal is to develop patient and mutation-specific test tools with patient derived in vitro “Twin” systems and humanized animal models for drug screenings and testing of potential genetic targeting strategies. RECORD offers young doctors a career development structure in the field of research and clinical care of patients with rare diseases. Scientists are supervised by two mentors and take part in the local Clinician Scientist Programs and are encouraged to participate in congresses, network meetings, seminars.
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