Progress made to improve gene therapy for treating disease without dose limiting side effects

For many disabling or fatal diseases, there is pre-clinical or clinical evidence of the potential therapeutic benefits of gene therapy. Unfortunately, the limitations of current gene transfer technologies have prevented successful trials or even led to serious adverse effects during trials.

The EU-funded project PERSIST ('Persisting transgenesis') was concerned with the development of new gene therapy tools and technologies for clinical application.

The project was successful on a number of levels. First, partners developed new strategies for long-term transgene expression while lowering the risk of induced immune responses, transgene toxicity and genotoxicity.

PERSIST thus overcomes the uncertainty related to dose-limiting side effects of earlier untargeted types of gene therapy. This work can now be applied towards the definition of safer and more effective treatment protocols for human diseases.

The results of the PERSIST project will contribute to improving treatment for the specific diseases the researchers studied. The research could also be applied to several other inherited diseases and sets the stage for applications in acquired diseases such as cancer and for infectious diseases.

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