UAB researchers have reversed metabolic dysfunction-associated steatohepatitis (MASH) in mouse models. MASH is a severe liver disease associated with obesity and type 2 diabetes that affects more than 40 million people. The results were obtained with a single intramuscular administration of the therapeutic viral vectors.
The research has also determined that most obese, type 2 diabetic and MASH patients could benefit from the therapy. The results will be the basis for a future clinical trial by the biopharmaceutical company Kriya ( https://kriyatherapeutics.com/ ).
Researchers from the Universitat Autonoma de Barcelona (UAB) in collaboration with clinicians from the Parc Taulí University Hospital in Sabadell have described in mice the long-term efficacy and safety of the intramuscular administration of a gene therapy for the treatment of MASH, a liver disease that affects approximately 40 million people in the United States and Europe. The therapy developed by the UAB researchers is based on the genetic engineering of the skeletal muscle with the gene that encodes for the fibroblast growth factor 21 protein using adeno-associated viral vectors (AAV-FGF21 vectors). FGF21 is a key metabolic regulator that, upon the administration of the vectors in skeletal muscle, is sustainedly increased in bloodstream (more than a year in this study).
This treatment mediates long-term reversal of liver fibrosis and MASH, counteracts obesity, excessive fat accumulation, insulin resistance ch.