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Development of gene therapy

inase deficiency and chronic granulomatous disease. There are also initial indicators that point to the efficacy of gene therapy in treating chronic lymphatic leukaemia and haemophilia B. But successes in clinical testing come with their share of setbacks, such as the currently slight margin of therapeutic effectiveness over unwanted side effects. This underscores the necessity to carefully assess risks and benefits and also shows that there is still a dire need for more research on gene therapy. Basic research should be conducted in direct collaboration across disciplines, using animal models and clinical studies. An acute need for research exists, especially regarding the development of efficient and safe vectors for gene transfer.


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Source:Deutsche Forschungsgemeinschaft


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