05/04/2011 VHIR's research shows it's possible to use gene therapy for MNGIE disease 05/04/2011 Researchers from Vall d'Hebron Research Institute (VHIR) have discovered a possible treatment for patients with the life-threatening minority disease MNGIE. The research group on neuromuscular and mitochondrial diseases, in collaboration with the gene therapy group at VHIR have been able, thanks to gene therapy, to introduce in an animal model the gene encoding the enzyme inactive in the disease, which has made it possible to reverse the biochemical imbalances characteristic of it. On the other hand, have discovered the biochemical mechanism responsible for the main dysfunction observed in patients, which opens new pharmacological treatment strategies that could slow the progression of the disease, while awaiting definitive treatment. Researchers from Vall d'Hebron Research Institute (VHIR) have discovered a possible treatment for patients with the life-threatening minority disease MNGIE. The research group on neuromuscular and mitochondrial diseases, in collaboration with the gene therapy group at VHIR have been able, thanks to gene therapy, to introduce in an animal model the gene encoding the enzyme inactive in the disease, which has made it possible to reverse the biochemical imbalances characteristic of it. On the other hand, have discovered the biochemical mechanism responsible for the main dysfunction observed in patients, which opens new pharmacological treatment strategies that could slow the progression of the disease, while awaiting definitive treatment. Twitter LinkedIn Facebook Whatsapp
