Publications
Information about journal articles, updated at 20-07-2025, from platform CIÊNCIAVITAE.
Exploring the potential of cell-derived vesicles for transient delivery of gene editing payloads
Leandro, Kevin; Rufino-Ramos, David; Breyne, Koen; Di Ianni, Emilio; Lopes, Sara M.; Jorge Nobre, Rui; Kleinstiver, Benjamin P.; et al, Advanced Drug Delivery Reviews. 211. 2024. https://doi.org/10.1016/j.addr.2024.115346 . 10.1016/j.addr.2024.115346 . published Advanced Drug Delivery Reviews
Regional distribution of polymorphisms associated to the disease-causing gene of spinocerebellar ataxia type 3
Elter, Tim Lukas; Sturm, Daniel; Santana, Magda M.; Schaprian, Tamara; Raposo, Mafalda; Melo, Ana Rosa Vieira; Lima, Manuela; et al, Journal of Neurology. 1. 272. 2024. https://doi.org/10.1007/s00415-024-12829-9 . 10.1007/s00415-024-12829-9 . published Journal of Neurology
Isolation of Adeno-associated Viral Vectors Through a Single-step and Semi-automated Heparin Affinity Chromatography Protocol
Miguel M. Lopes; Lopes, Sara M. ; Rafael Baganha; Carina Henriques; Ana C. Silva; Diana D. Lobo; Luísa Cortes; Luís Pereira de Almeida; Rui Jorge Nobre, Journal of Visualized Experiments (JoVE). 206. 2024. published Journal of Visualized Experiments (JoVE)
A new protocol for whole-brain biodistribution analysis of AAVs by tissue clearing, light-sheet microscopy and semi-automated spatial quantification
Miguel M. Lopes; Jacques Paysan; José Rino; Sara M. Lopes; Pereira de Almeida, Luis ; Luísa Cortes; Rui Jorge Nobre, Gene Therapy. 2022. https://doi.org/10.1038/s41434-022-00372-z . 10.1038/s41434-022-00372-z . Gene Therapy
ULK overexpression mitigates motor deficits and neuropathology in mouse models of Machado-Joseph disease
Vasconcelos-Ferreira, Ana; Martins, Inês Morgado; Lobo, Diana; Pereira, Dina; Miguel M. Lopes; Faro, Rosário; Lopes, Sara M. ; et al, Molecular Therapy. 370 - 387. 1. 30. 2022. http://dx.doi.org/10.1016/j.ymthe.2021.07.012 . 10.1016/j.ymthe.2021.07.012 . published Molecular Therapy
MiRNA-Mediated Knockdown of ATXN3 Alleviates Molecular Disease Hallmarks in a Mouse Model for Spinocerebellar Ataxia Type 3
Nobre, Rui Jorge; Lobo, Diana D.; Henriques, Carina; Duarte, Sonia P.; Lopes, Sara M. ; Silva, Ana C.; Miguel M. Lopes; et al, Nucleic Acid Therapeutics. 2021. http://dx.doi.org/10.1089/nat.2021.0020 . 10.1089/nat.2021.0020 . published Nucleic Acid Therapeutics
Protocol for the Characterization of the CAG Tract and Flanking Polymorphisms in Machado-Joseph Disease: Impact on Diagnosis and Development of Gene-Based Therapies
Lopes, Sara Monteiro; Faro, Rosário; Miguel M. Lopes; Onofre, Isabel; Mendonça, Nuno; Ribeiro, Joana; Januário, Cristina; Nobre, Rui Jorge; Pereira de Almeida, Luis , The Journal of Molecular Diagnostics. 2020. http://dx.doi.org/10.1016/j.jmoldx.2020.03.003 . 10.1016/j.jmoldx.2020.03.003 . The Journal of Molecular Diagnostics
Antisense oligonucleotide therapeutics in neurodegenerative diseases: the case of polyglutamine disorders
Silva, Ana C; Lobo, Diana D; Martins, Inês M; Lopes, Sara Monteiro; Henriques, Carina; Duarte, Sónia P; Dodart, Jean-Cosme; Nobre, Rui Jorge; Pereira de Almeida, Luis , Brain. 407 - 429. 2. 143. 2020. http://dx.doi.org/10.1093/brain/awz328 . 10.1093/brain/awz328 . published Brain
Fibroblasts of Machado Joseph Disease patients reveal autophagy impairment
Onofre, Isabel; Mendonça, Nuno; Lopes, Sara; Nobre, Rui; de Melo, Joana Barbosa; Carreira, Isabel Marques; Januário, Cristina; Gonçalves, António Freire; Pereira de Almeida, Luis , Scientific Reports. 1. 6. 2016. http://dx.doi.org/10.1038/srep28220 . 10.1038/srep28220 . published Scientific Reports
5 9 out of 9 Publications
