| 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 | 14 | 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, SD NC, ... Medicina 79 (6), 493-501, 2019 | 8* | 2019 |
| 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 |
| 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 |
| A Novel Gene Therapy for Rett Syndrome through Reactivation of the Silent X Chromosome S Powers, S Lou, S Nardini, S Likhite, J Delgado, A Sierra, M Baird, ... MOLECULAR THERAPY 30 (4), 395-395, 2022 | | 2022 |
| In vitro modeling for neurological diseases using direct conversion from fibroblasts to neuronal progenitor cells and differentiation into astrocytes RS Sinha, R Yacidzohara, M Kathrin | | 2021 |
| Rapid reprogramming of ALS patient fibroblasts differentiates CuATSM responders from nonresponders. C Dennys, F Roussel, X Zhang, R Rodrigo, A Hartlaub, A Sierra-Delgado, ... MDPI, 2020 | | 2020 |
| Multi-Center Blinded Preclinical Efficacy Study Shows Significant Differences in Promoter Performance for Gene Replacement Therapy of SMARD1/CMT2S JAS Delgado, S Likhite, V McGovern, A Huffenberger, D Chugh, AN Hicks, ... MOLECULAR THERAPY 28 (4), 4-4, 2020 | | 2020 |
| Neutralizing Antibodies Against Selected AAV Serotypes in a Colombian Population P Katherine Bautista-Nino, JA Sierra-Delgado, S Likhite, LE Echeverria, ... MOLECULAR THERAPY 28 (4), 183-184, 2020 | | 2020 |
| Prevalence of Neutralizing Antibodies against Adeno-Associated Virus Serotypes 1, 2, and 9 in Non-Injected Latin American Patients with Heart Failure JA Sierra, S Likhite, LE Echeverria, E Guio, C Vargas, NC Serrano, ... Molecular Therapy 26 (5), 107-107, 2018 | | 2018 |
