| Identification of ezetimibe and pranlukast as pharmacological chaperones for the treatment of the rare disease mucopolysaccharidosis type IVA CJ Alméciga-Diaz, OA Hidalgo, S Olarte-Avellaneda, A Rodríguez-López, ... Journal of medicinal chemistry 62 (13), 6175-6189, 2019 | 35 | 2019 |
| Neonatal nonviral gene editing with the CRISPR/Cas9 system improves some cardiovascular, respiratory, and bone disease features of the mucopolysaccharidosis I phenotype in mice RS Schuh, EA Gonzalez, AMV Tavares, BG Seolin, LS Elias, LNP Vera, ... Gene Therapy 27 (1), 74-84, 2020 | 32 | 2020 |
| Human recombinant lysosomal enzymes produced in microorganisms ÁJ Espejo-Mojica, CJ Alméciga-Díaz, A Rodríguez, Á Mosquera, D Díaz, ... Molecular genetics and metabolism 116 (1-2), 13-23, 2015 | 31 | 2015 |
| Production and characterization of a human lysosomal recombinant iduronate‐2‐sulfatase produced in Pichia pastoris N Pimentel, A Rodríguez‐Lopez, S Díaz, JC Losada, DJ Díaz‐Rincón, ... Biotechnology and applied biochemistry 65 (5), 655-664, 2018 | 20 | 2018 |
| Characterization of human recombinant N-acetylgalactosamine-6-sulfate sulfatase produced in Pichia pastoris as potential enzyme for mucopolysaccharidosis IVA treatment A Rodríguez-López, LN Pimentel-Vera, AJ Espejo-Mojica, A Van Hecke, ... Journal of Pharmaceutical Sciences 108 (8), 2534-2541, 2019 | 17 | 2019 |
| Evidence for inflammasome activation in the brain of mucopolysaccharidosis type II mice AS Azambuja, LN Pimentel-Vera, EA Gonzalez, E Poletto, CV Pinheiro, ... Metabolic Brain Disease 35, 1231-1236, 2020 | 16 | 2020 |
| Improved engraftment and therapeutic efficacy by human genome-edited hematopoietic stem cells with Busulfan-based myeloablation E Poletto, P Colella, LNP Vera, S Khan, S Tomatsu, G Baldo, ... Molecular Therapy-Methods & Clinical Development 25, 392-409, 2022 | 15 | 2022 |
| Brain and visceral gene editing of mucopolysaccharidosis I mice by nasal delivery of the CRISPR/Cas9 system LNP Vera, RS Schuh, FNS Fachel, E Poletto, E Piovesan, F Kubaski, ... The Journal of Gene Medicine 24 (4), e3410, 2022 | 13 | 2022 |
| Virtual screening of potential inhibitors for SARS-CoV-2 main protease CJ Alméciga-Díaz, LN Pimentel-Vera, A Caro, A Mosquera, ... MDPI AG, 2020 | 13 | 2020 |
| Identification of the iduronate-2-sulfatase proteome in wild-type mouse brain C Cardona, E Benincore, N Pimentel, LH Reyes, C Patarroyo, ... Heliyon 5 (5), 2019 | 9 | 2019 |
| Evaluation of oxidative stress and mitochondrial function in a type II mucopolysaccharidosis cellular model: in vitro effects of genistein and coenzyme Q10 CED Jacques, FF Lopes, E Poletto, LNP Vera, P Vianna, LS Reinhardt, ... Metabolic Brain Disease 38 (2), 519-529, 2023 | 7 | 2023 |
| The potential of gene therapy for mucopolysaccharidosis type I LNP Vera, G Baldo Expert Opinion on Orphan Drugs 8 (1), 33-41, 2020 | 7 | 2020 |
| In situ gene therapy HS Santos, L Rodrigues, LNP Vera, E Poletto, E Filippi-Chiela, ... Current Gene Therapy 21 (5), 406-430, 2021 | 5 | 2021 |
| CNS-wide repopulation by hematopoietic-derived microglia-like cells corrects progranulin deficiency in mice P Colella, R Sayana, MV Suarez-Nieto, J Sarno, K Nyame, J Xiong, ... Nature Communications 15 (1), 5654, 2024 | 2 | 2024 |
| Cell uptake evaluation of human recombinant lysosomal enzymes produced in Pichia pastoris CJ Almeciga, A Espejo-Mojica, A Rodriguez-López, C Losada, J Sanchez, ... Molecular Genetics and Metabolism 2 (117), S17-S18, 2016 | 2 | 2016 |
| Effect of genistein and coenzyme Q10 in oxidative damage and mitochondrial membrane potential in an attenuated type II mucopolysaccharidosis cellular model CA Delgado, E Poletto, LNP Vera, CED Jacques, P Vianna, LS Reinhardt, ... Cell Biochemistry and Function 42 (2), e3932, 2024 | 1 | 2024 |
| Comparison of different DNA preservation solutions for oral cytological samples JS Nunes, LN Pimentel-Vera, SB Silva, B de Bem Prunes, PV Rados, ... Archives of Oral Biology 158, 105867, 2024 | 1 | 2024 |
| CNS Repopulation by Hematopoietic-Derived Microglia-Like Cells Corrects Progranulin deficiency P Colella, R Sayana, MV Suarez-Nieto, J Sarno, K Nyame, J Xiong, ... Research Square, 2023 | 1 | 2023 |
| Edição gênica em mucopolissacaridose tipos I e II utilizando o sistema CRISPR-Cas9: uma abordagem não-invasiva para o tratamento do comprometimento neurológico e sistêmico LNP Vera | 1 | 2021 |
| Genome editing in lysosomal disorders LN Pimentel-Vera, E Poletto, EA Gonzalez, F de Oliveira Poswar, ... Progress in Molecular Biology and Translational Science 182, 289-325, 2021 | 1 | 2021 |
