EXTL2 and EXTL3 inhibition with siRNAs as a promising substrate reduction therapy for Sanfilippo C syndrome I Canals, N Benetó, M Cozar, L Vilageliu, D Grinberg Scientific reports 5 (1), 13654, 2015 | 35 | 2015 |
Sanfilippo syndrome: molecular basis, disease models and therapeutic approaches N Benetó, L Vilageliu, D Grinberg, I Canals International Journal of Molecular Sciences 21 (21), 7819, 2020 | 31 | 2020 |
Therapeutic strategies targeting mitochondrial calcium signaling: a new hope for neurological diseases? LR Rodríguez, T Lapeña-Luzón, N Benetó, V Beltran-Beltran, FV Pallardó, ... Antioxidants 11 (1), 165, 2022 | 20 | 2022 |
Neuronal and astrocytic differentiation from Sanfilippo C syndrome iPSCs for disease modeling and drug development N Benetó, M Cozar, L Castilla-Vallmanya, OG Zetterdahl, M Sacultanu, ... Journal of Clinical Medicine 9 (3), 644, 2020 | 13 | 2020 |
Generation of two compound heterozygous HGSNAT-mutated lines from healthy induced pluripotent stem cells using CRISPR/Cas9 to model Sanfilippo C syndrome N Benetó, M Cozar, M García-Morant, E Creus-Bachiller, ... Stem Cell Research, 2019, vol. 41, num. 101616, p. 1-5, 2019 | 10 | 2019 |
Generation of two NAGLU-mutated homozygous cell lines from healthy induced pluripotent stem cells using CRISPR/Cas9 to model Sanfilippo B syndrome N Benetó, M Cozar, L Gort i Mas, L Pacheco, L Vilageliu i Arqués, ... Stem Cell Research, 2020, vol. 42, p. 101668, 2020 | 7 | 2020 |
Cofilin and Neurodegeneration: New Functions for an Old but Gold Protein T Lapeña-Luzón, LR Rodríguez, V Beltran-Beltran, N Benetó, FV Pallardó, ... Brain sciences 11 (7), 954, 2021 | 6 | 2021 |
Role of adenosine receptors in rare neurodegenerative diseases with motor symptoms V Beltran-Beltran, N Beneto, T Lapeña-Luzón, LR Rodriguez, FV Pallardó, ... Current Protein and Peptide Science 22 (9), 675-694, 2021 | 1 | 2021 |
DDC expression is not regulated by NFAT5 (TonEBP) in dopaminergic neural cell lines L Pineda-Cirera, J Cabana-Domínguez, N Benetó, H Diez, C Arenas, ... Gene 742, 144569, 2020 | 1 | 2020 |
MIN-102 enhance the oxidative profile of FRDA mouse model T Lapena-Luzon, N Beneto, L Rodriguez-Pascau, P Pizcueta, C Vergara, ... FEBS OPEN BIO 12, 127-127, 2022 | | 2022 |
Deciphering frataxin implication in endoplasmic reticulum-mitochondria associated membranes (MAMs) LR Rodriguez, D Pla-Martin, A Lerma-Aguilera, T Lapena-Luzon, ... FEBS OPEN BIO 12, 21-21, 2022 | | 2022 |
Genome Editing Using Cas9-gRNA Ribonucleoprotein in Human Pluripotent Stem Cells for Disease Modeling N Benetó, D Grinberg, L Vilageliu, I Canals Induced Pluripotent Stem Cells and Human Disease: Methods and Protocols, 409-425, 2021 | | 2021 |
DDC expression is not regulated by NFAT5 (TonEBP) in dopaminergic neural cell lines upon hypertonic stress L Pineda-Cirera, J Cabana Domínguez, N Benetó, H Díeze, ... Gene, 2020, vol. 742, 2020 | | 2020 |
Novel neuronal and astrocytic models for Sanfilippo C syndrome using CRISPR/Cas9-edited iPSC. Their use in therapeutic approaches N Beneto, M Cozar, M Garcia-Morant, E Creus-Bachiller, D Grinberg, ... HUMAN GENE THERAPY 30 (11), A150-A150, 2019 | | 2019 |
Substrate reduction therapy approach for Sanfilippo C syndrome: use of iPSC and iPSC-derived neurons from patients as cellular models N Beneto, E Creus-Bachiller, M Garcia-Morant, D Grinberg, L Vilageliu, ... EUROPEAN JOURNAL OF HUMAN GENETICS 27, 859-859, 2019 | | 2019 |
EXTL2 and EXTL3 inhibition with siRNAs as a promising substrate reduction therapy for Sanfilippo C syndrome I Canals Montferrer, N Benetó, M Cozar, L Vilageliu i Arqués, ... Scientific Reports, 2015, vol. 5, p. 13654, 2015 | | 2015 |