| CuATSM effectively ameliorates ALS patient astrocyte‐mediated motor neuron toxicity in human in vitro models of amyotrophic lateral sclerosis CN Dennys, F Roussel, R Rodrigo, X Zhang, A Sierra Delgado, ... Glia 71 (2), 350-365, 2023 | 15 | 2023 |
| In vitro modeling for neurological diseases using direct conversion from fibroblasts to neuronal progenitor cells and differentiation into astrocytes CN Dennys, JA Sierra-Delgado, SS Ray, AM Hartlaub, FS Roussel, ... Journal of Visualized Experiments (JoVE), e62016, 2021 | 11 | 2021 |
| Immune response and gene therapy with adenoassociated viral vectors JA Sierra-Delgado, PK Bautista-Nino, CI Vargas-Castellanos, NCS Diaz, ... Medicina 79 (6), 493-501, 2019 | 8* | 2019 |
| On certain coverings of generalized polygons A Delgado, R Weiss Bulletin of the London Mathematical Society 21 (3), 235-242, 1989 | 8 | 1989 |
| Prevalence of neutralizing antibodies against adeno-associated virus serotypes 1, 2, and 9 in non-injected latin american patients with heart failure—ANVIAS study JA Sierra-Delgado, S Likhite, PK Bautista, SA Gómez-Ochoa, ... International journal of molecular sciences 24 (6), 5579, 2023 | 7 | 2023 |
| In vitro modeling as a tool for testing therapeutics for spinal muscular atrophy and IGHMBP2-related disorders JA Sierra-Delgado, S Sinha-Ray, A Kaleem, M Ganjibakhsh, M Parvate, ... Biology 12 (6), 867, 2023 | 6 | 2023 |
| RNA helicase IGHMBP2 regulates THO complex to ensure cellular mRNA homeostasis AB Prusty, A Hirmer, JA Sierra-Delgado, H Huber, UP Guenther, ... Cell Reports 43 (2), 2024 | 5 | 2024 |
| In-Depth Comparison of Anc80L65 and AAV9 Retinal Targeting and Characterization of Cross-Reactivity to Multiple AAV Serotypes in Humans MK Schwartz, S Likhite, TA Vetter, MC Baird, V McGovern, ... Molecular Therapy-Methods & Clinical Development, 2023 | 5 | 2023 |
| MeCP2 gene therapy ameliorates disease phenotype in mouse model for Pitt Hopkins syndrome CN Dennys, SAD Vermudez, RJM Deacon, JA Sierra-Delgado, K Rich, ... Neurotherapeutics 21 (5), e00376, 2024 | 3 | 2024 |
| MECHANISMS FOR THE EPIGENETIC REGULATION OF MITOCHONDRIAL DNA JA SIERRA-DELGADO, GA CONTRERAS-GARCÍA BAG-JOURNAL OF BASIC AND APPLIED GENETICS Учредители: Socieded Argentina de …, 2016 | 2* | 2016 |
| Use of bioinformatic tools in primer validation JA Sierra-Delgado, VL Pérez, CI Vargas Genetics in meDicine 18 (8), 855-856, 2016 | 1 | 2016 |
| Kaempferol enhances ER-mitochondria coupling and protects motor neurons from mitochondrial dysfunction and ER stress in C9ORF72-ALS F Pilotto, PH Smeele, O Scheidegger, R Diab, M Schobesberger, ... Acta Neuropathologica Communications 13 (1), 21, 2025 | | 2025 |
| Kaempferol enhances ER-mitochondria coupling and protects motor neurons from ER stress and mitochondrial dysfunction in C9ORF72-ALS F Pilotto, P Smeele, O Scheidegger, R Diab, M Schobesberger, ... | | 2024 |
| 471P Interfering with CUG toxic repeats using AAV. U7snRNAs rescue myotonia and splicing defects in myotonic dystrophy type 1 C Almeida, A Brinkman, C Wendt, A Blatnik, A Delgado, L Gushchina, ... Neuromuscular Disorders 43, 104441.546, 2024 | | 2024 |
| The miREX Platform Technology Allows for Highly Efficacious X-Reactivation for Treatment of X-linked Disorders C Dennys, SL Powers, M Khani, S Lou, SB Likhite, L Sass, AS Delgado, ... MOLECULAR THERAPY 32 (4), 317-318, 2024 | | 2024 |
| BIUXX M Ganjibakhsh, JAS Delgado, B Ogunlade, Y Tkachenko, A Kaleem, ... MOLECULAR THERAPY 32 (4), 542-543, 2024 | | 2024 |
| Correction of Myotonia and Reduction of Toxic DMPK Foci After Treatment of the HSALR Mouse Model of Myotonic Dystrophy Type 1 Using an Adeno-Associated Virus Delivering U7snRNA … C Almeida, A Brinkman, AS Delgado, LV Gushchina, D Arnold, R Weiss, ... MOLECULAR THERAPY 32 (4), 216-216, 2024 | | 2024 |
| Novel AAV Gene Therapy Candidates Show Promise In Vitro and In Vivo for Treatment of PGAP3-CDG Syndrome JAS Delgado, M Simon, R Talekar, X Zhang, A Kaleem, M Ganjibakhsh, ... MOLECULAR THERAPY 32 (4), 316-316, 2024 | | 2024 |
| Multicenter AAV Gene Therapy Studies for SMARD1/CMT2S Establish Safety and Efficacy in Multiple Animal Models and Pave the Way for Initiation of a Phase I/II Clinical Trial JAS Delgado, S Likhite, SE Holbrook, V McGovern, D Chung, J Caporale, ... MOLECULAR THERAPY 30 (4), 17-18, 2022 | | 2022 |
| Evaluation of AAV9 Gene Therapy for SMARD1/CMT2S in Different Mouse Models Reveal Differences in Efficacy Dependent on Promoter Choice JAS Delgado, S Likhite, V McGovern, S Holbrook, A Huffenberger, ... MOLECULAR THERAPY 30 (4), 500-501, 2022 | | 2022 |
