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Since living memory, the basic treatment strategies for the therapy of human diseases has not much changed conceptually. Although the molecular understanding of metabolic disorders continues to progress significantly the therapeutic strategy is still based on specific, often heterologous compounds which interfere with critical disease targets, trigger a metabolic bypass reaction or complement a mo ...

"For many disabling or fatal diseases, there is pre-clinical or clinical evidence of the potential therapeutic efficacy of gene therapy and, yet, the limitations of current gene transfer technologies have prevented success or even caused serious adverse events leading to termination of trials. PERSIST will explore the use of highly innovative gene-modifying and delivery technologies and capitalize ...