Dtsch Arztebl Int
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Dtsch Arztebl Int
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New gene therapy approaches have emerged as promising treatment options for rare congenital disorders and certain tumor entities for which previously only procedures of limited curative potential had been available, if at all. ⋯ The first gene treatments have now been approved and bring hope of long-term therapeutic benefit after a single administration. The numbers of patients who come into question for specific therapies are often low, so that many different aspects- generation of evidence on efficacy and safety, determining indications, performance of the treatment, pricing-bring new challenges.