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.

The PERSIST project had four main goals which are strategic for innovative technology development in the field of genetic engineering for persisting gene expression:

– Long-term gene transfer without adverse effect on the host chromatin, including stable non-integrative gene transfer and targeted integration without impact on endogenous gene transcription,

– Site-specific genome modification, allowing a range of approaches such as gene correction, gene targeting and site-specific gene addition,

– Tight control on target cell selection or immune recognition by engineering the vector particle,

– Tight control of transgene expression exploiting recent discoveries on gene expression control, such as microRNA, on chromatin structure and organisation, and designer transcriptional switches.

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