Therapeutic advantages of combined gene/cell therapy strategies in a murine model of GM2 gangliosidosis
Davide Sala, Francesca Ornaghi, Francesco Morena, Chiara Argentati, Manuela Valsecchi, Valeria Alberizzi, Roberta Di Guardo, Alessandra Bolino, Massimo Aureli, Sabata Martino, 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.
Molecular Therapy — Methods & Clinical Development · Mar 16, 2022Peer-reviewedGenetics