| CRISPR/Cas9 editing for gaucher disease modelling E Pavan, M Ormazabal, P Peruzzo, E Vaena, P Rozenfeld, A Dardis International journal of molecular sciences 21 (9), 3268, 2020 | 18 | 2020 |
| Bone marrow adipocytes alteration in an in vitro model of Gaucher disease A Crivaro, JM Mucci, C Bondar, M Ormazabal, E Vaena, MV Delpino, ... Molecular Genetics and Metabolism Reports 36, 100980, 2023 | | 2023 |
| Exploring the Pathophysiologic Cascade Leading to Osteoclastogenic Activation in Gaucher Disease Monocytes Generated via CRISPR/Cas9 Technology ME Ormazabal, E Pavan, E Vaena, D Ferino, J Biasizzo, JM Mucci, ... International Journal of Molecular Sciences 24 (13), 11204, 2023 | | 2023 |
| Lyso-Gb3 determination in classic and late onset Fabry patients PA Rozenfeld, R Ceci, A Velazco, E Vaena, AN Crivaro, JM Mucci, ... Molecular Genetics and Metabolism 138 (2), 107299, 2023 | | 2023 |
| Generation of an in vitro model for Hunter syndrome using CRISPR/Cas9 technology E Vaena, ME Ormazabal, E Pavan, JM Mucci, AN Crivaro, C Bondar, ... Molecular Genetics and Metabolism 138 (2), 107339, 2023 | | 2023 |
| Expression of caspase-3 and TGF-β1 in Fabry disease biopsies: Correlation with clinical parameters C Bondar, JM Mucci, AN Crivaro, E Vaena, ME Ormazabal, PA Rozenfeld Molecular Genetics and Metabolism 138 (2), 107033, 2023 | | 2023 |
