GeneticsMolecular Therapy — Methods & Clinical DevelopmentPeer-reviewed
Therapeutic advantages of combined gene/cell therapy strategies in a murine model of GM2 gangliosidosis
Davide Sala, Francesca Ornaghi, Francesco Morena…·Angela Gritti
This study shows that combining gene therapy and bone marrow transplantation in a mouse model of GM2 gangliosidosis improves lifespan and reduces disease symptoms more effectively than either treatment alone.
5 citations