| Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy V Haurigot, S Marcó, A Ribera, M Garcia, A Ruzo, P Villacampa, E Ayuso, ... The Journal of clinical investigation 123 (8), 3254-3271, 2013 | 245 | 2013 |
| FGF21 gene therapy as treatment for obesity and insulin resistance V Jimenez, C Jambrina, E Casana, V Sacristan, S Muñoz, S Darriba, ... EMBO molecular medicine 10 (8), e8791, 2018 | 234 | 2018 |
| Correction of pathological accumulation of glycosaminoglycans in central nervous system and peripheral tissues of MPSIIIA mice through systemic AAV9 gene transfer A Ruzo, S Marcó, M García, P Villacampa, A Ribera, E Ayuso, L Maggioni, ... Human gene therapy 23 (12), 1237-1246, 2012 | 136 | 2012 |
| CNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome) S Motas, V Haurigot, M Garcia, S Marcó, A Ribera, C Roca, X Sánchez, ... JCI insight 1 (9), 2016 | 89 | 2016 |
| Liver production of sulfamidase reverses peripheral and ameliorates CNS pathology in mucopolysaccharidosis IIIA mice A Ruzo, M Garcia, A Ribera, P Villacampa, V Haurigot, S Marcó, E Ayuso, ... Molecular Therapy 20 (2), 254-266, 2012 | 70 | 2012 |
| Biochemical, histological and functional correction of mucopolysaccharidosis type IIIB by intra-cerebrospinal fluid gene therapy A Ribera, V Haurigot, M Garcia, S Marcó, S Motas, P Villacampa, ... Human molecular genetics 24 (7), 2078-2095, 2015 | 67 | 2015 |
| Disease correction by AAV-mediated gene therapy in a new mouse model of mucopolysaccharidosis type IIID C Roca, S Motas, S Marcó, A Ribera, V Sánchez, X Sánchez, J Bertolin, ... Human molecular genetics 26 (8), 1535-1551, 2017 | 54 | 2017 |
| In Vivo Gene Therapy for Mucopolysaccharidosis Type III (Sanfilippo Syndrome): A New Treatment Horizon S Marcó, V Haurigot, F Bosch Human gene therapy 30 (10), 1211-1221, 2019 | 44 | 2019 |
| Progressive neurologic and somatic disease in a novel mouse model of human mucopolysaccharidosis type IIIC S Marcó, A Pujol, C Roca, S Motas, A Ribera, M Garcia, M Molas, ... Disease models & mechanisms 9 (9), 999-1013, 2016 | 29 | 2016 |
| Seven-year follow-up of durability and safety of AAV CNS gene therapy for a lysosomal storage disorder in a large animal S Marcó, V Haurigot, ML Jaén, A Ribera, V Sánchez, M Molas, M Garcia, ... Molecular Therapy-Methods & Clinical Development 23, 370-389, 2021 | 25 | 2021 |
| Treatment of skeletal and non-skeletal alterations of Mucopolysaccharidosis type IVA by AAV-mediated gene therapy J Bertolin, V Sánchez, A Ribera, ML Jaén, M Garcia, A Pujol, X Sánchez, ... Nature communications 12 (1), 1-14, 2021 | 17 | 2021 |
| ESGCT XXV Anniversary Congress in Collaboration with the German Society for Gene Therapy October 17–20, 2017 Berlin, Germany SM Maffioletti, S Sarcar, A Henderson, I Mannhardt, L Pinton, LA Moyle, ... Human Gene Therapy 28 (12), A1-A125, 2017 | 2 | 2017 |
| European Society of Gene and Cell Therapy French Society of Cell and Gene Therapy Collaborative Congress 2012 October 25–29, 2012 Palais des Congrès de Versailles Versailles … T VandenDriessche, RA Pearson, RJ Chandler, CC Bartholomae, ... Human Gene Therapy 23 (10), A1-A173, 2012 | 2 | 2012 |
| Safe and sustained elevation of sulfamidase after administration of AAV9-sulfamidase to the CSF of dogs: Seven year-follow up S Marco, V Haurigot, ML Jaen, A Ribera, V Sanchez, M Garcia, S Motas, ... HUMAN GENE THERAPY 30 (11), A93-A93, 2019 | 1 | 2019 |
| AAV-Mediated Sulfamidase Expression in the Liver Prevents Development of Somatic Alterations in MPS IIIA Mice A Ruzo, M Garcia, V Haurigot, S Marco, P Villacampa, A Ribera, C Roca, ... Human Gene Therapy 20 (9), 1045-1046, 2009 | 1 | 2009 |
| AAV-Mediated Gene Therapy to Treat Mucopolysaccharidosis Type IVA in a New Morquio a Mouse Model V Sanchez, J Bertolin, A Ribera, X Sanchez, S Marco, S Motas, J Perez, ... MOLECULAR THERAPY 28 (4), 492-492, 2020 | | 2020 |
| Widespread CNS Sulfamidase Expression Seven Years after AAV9-Mediated Gene Transfer to the CSF of Dogs S Marco, V Haurigot, A Ribera, V Sanchez, M Luisa Jaen, M Molas, ... MOLECULAR THERAPY 28 (4), 216-216, 2020 | | 2020 |
| Generation of a rat model of Mucopolysaccharidosis type IVA (Morquio A disease) that reproduces the human pathology J Bertolin, A Pujol, M Garcia, V Sanchez, A Ribera, ML Jaen, S Munoz, ... HUMAN GENE THERAPY 30 (11), A158-A158, 2019 | | 2019 |
| AAV-mediated gene therapy to treat mucopolysaccharidosis type IVA in a new mouse model of the disease V Sanchez, J Bertolin, A Ribera, X Sanchez, S Marco, S Motas, J Perez, ... HUMAN GENE THERAPY 30 (11), A172-A172, 2019 | | 2019 |
| Biochemical, histological and behavioural characterization of a new mouse model of Niemann-Pick C2 disease X Sanchez, G Elias, A Ribera, V Sanchez, J Bertolin, J Perez, C Roca, ... HUMAN GENE THERAPY 30 (11), A157-A157, 2019 | | 2019 |
