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The Faculty of the Gene Therapy Center is dedicated developing therapeutic approaches for rare inherited diseases for which there is no cure utilizing state of the art technologies to correct the genetic mutations.
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Gene Therapy holds the promise to treat rare genetic disorders with precision medicine alleviating the suffering of those that are hit by misfortune by a personalized medicine approach.
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Recombinant Adeno-associated viruses are safe vectors to introduce genes into cells. Different serotypes allow for tissue specific infection, reducing unwanted side effects of the therapeutic treatment.
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