Three Genethon experts are participating in the MAGIC project (Next-generation Models and Genetic therapIes for rare neuromusCular diseases), dedicated to the development of innovative therapeutic strategies in muscular dystrophies.
Led by Prof. Francesco Saverio Tedesco (University College London / Francis Crick Institute), with the support of Mario Amendola (Inserm/Genethon), the MAGIC project gathers 15 partners from 9 countries with a primary objective: to accelerate the development of innovative gene therapy approaches and genome editing strategies for muscular dystrophies. It is funded by Horizon Europe and UK Research and Innovation.
Genethon researchers – Mario Amendola, (Inserm), director of” therapeutic genome editing” group, Giuseppe Ronzitti, (Inserm), director of the “Immunology and Liver Diseases” group and David-Alexandre Gross, Inserm researcher associated with the “Immunology and Liver Diseases” group – will bring their expertise and contribute to the Magic project at multiple levels:
- by developing novel genome editing strategies to treat Duchenne muscular dystrophy and optimizing editing tools for in vivo delivery.
- by identifying and creating novel tissue specific promoters for efficient and specific skeletal muscle and heart tissue expression.
- by studying the adaptive immune responses against the new viral vectors and transgenes, its impact on treatment efficacy as well as by establishing appropriate immunomodulation.