The technology we participate to develop, called "Zinc-Finger Artificial Transcription Factors (ZF-ATFs)", allows to design, realize and select artificial genes coding for proteins capable of recognizing and binding "potentially" any DNA sequence. We used ZF-ATF technology to reprogram the expression of "beneficial" genes capable of efficiently counteracting the negative effect of mutated genes related to rare diseases. In addition, we have combined ZF-ATF technology with adeno-associated vector (rAAV) technology to develop gene therapy projects applicable to Duchenne Muscular Dystrophy (DMD), Merosinopathy (MDC1A), other neuromuscular pathologies and various genetic or viral human pathologies.
The therapeutic strategy we propose specifically for Duchenne Muscular Dystrophy (DMD) is based on artificial transcription factors Zinc Finger (ZF-ATF), addresed to the "A" promoter of the utrophin gene to up-regulate its expression. The main advantages of our ZF-ATFs over other gene therapy technologies for DMD are; i) ZF-ATFs are applicable to all DMD patients regardless of the type of mutation present in Dystrophin; ii) ZF-ATFs are active at low concentrations and due to their small size they are ideal in gene therapy; iii) ZF-ATFs are a valid alternative to the replacement of large mutated genes; iv) ZF-ATFs mimic the natural mechanism of transcription regulation, producing all the "isoforms" of the target gene product; and v) Our ZF-ATFs are designed to avoid/reduce any immune response.
Italy, EPO, USA