Immunomediated Diseases and Innovative Therapies

We have developed a gene editing strategy targeting liver cells to treat mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) using a murine model of this metabolic disease. The delivery of the gene editing tools (based on CRISPR/Cas9) is based on nanoparticles that are being made in collaboration with the group of S. Borrós (Institut Químic de Sarrià) and the Canadian company Acuitas^ã. Also, in collaboration with the group of F. Munell (Pediatric Neurology, VHIR) and R. Hernández (University of Navarra) we are developing a novel gene therapy approach to treat congenital muscular dystrophy type 1A (MCD1A), a progressive and orphan genetic disease which represents about 40% of all congenital muscular dystrophies. Also, in collaboration with the group of J. Bueren (CIEMAT, Madrid), we have demonstrated the lack of immunotoxicity in an European gene therapy clinical trial for patients with Fanconi anemia A. Finally, in collaboration with the groups of J. Verdaguer (UdL) and J Gavaldà (VHIR), we are investigating the cytolytic and antimicrobial activity of a new family of peptides derived from intermediate filaments.