Artificial genes as a therapeutic strategy for muscular dystrophies

# Record card
70
Thematic areas
Health & Biotech / Bio-medicals
Health & Biotech / New therapies
Description

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.

Business fields
Industry
Type of innovation
Product innovation
Description of innovative features / Competitive advantages

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.

Reference market
Total innovation
Impacts on existing markets
Development stage
Prototype
TRL
4
5
Advantages
New product/process/service/technology
Patented technology
Yes
Country/ies

Italy, EPO, USA

Technology validation/demonstration
Internal validation
Market positioning
Italian
European
International
Partner required
Enteprise
Seed capital
Cooperation in national /european / international project

Information
For more information and/or to be put in contact with the Research Team, please contact the Project Manager:

Barbara Angelini - Project Manager
CNR - Unità Valorizzazione della Ricerca
Phone number 06.49932415
E-mail barbara.angelini@cnr.it