What's the Problem?

Genetic disorders are pathologies that are caused by gene abnormalities – such as the absence of a gene or a harmful mutation. The definitive treatment for these disorders is Gene Therapy, which consists in introducing a functional gene to the affected cells of a patient to recover lost functions. Suitable vehicles to deliver these genes are paramount for this process. Currently, viral vectors are the most widely used vehicles. The development of Gene Therapies is limited by these vectors because of problems such as: (1) Eligibility: not all the patients are eligible for these treatments. (2) Re-treatments: re-treatments are not possible. (3) Low specificity: the therapies have to be applied locally because of their very low tissue-specificity. (4) Payload: viral payload is limited, and so is the availability of treatments. (5) No versatility: if the gene or the target cell is changed, each vehicle must be synthesized from scratch.