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TITLE: AAV-mediated allele-specific silencing alleviates neuropathology in a novel non-human primate model of Spinocerebellar ataxia type 3
AUTHORS: Carina Henriques; Diana Duarte Lobo; Ana Carolina Silva; Ana Rita Fernandes; Miguel Monteiro Lopes; Audrey Fayard; Caroline Jan; Sophie Lecourtois; Martine Guillermier; Julien Flament; João Castelhano; Miguel Castelo-Branco; Rui Caetano Oliveira; Philippe Hantraye; Padmaja Yalamanchili; Steven de Marco; Romina Aron Badin; Luís Pereira de Almeida ; Rui Jorge Nobre;
PUBLISHED: 2025
INDEXED IN: CrossRef
2
TITLE: Autophagy- and oxidative stress-related protein deregulation mediated by extracellular vesicles of human MJD/SCA3 iPSC-derived neuroepithelial stem cells and differentiated neural cultures  Full Text
AUTHORS: Mendonca, Liliana S.; Moreira, Ricardo; Henriques, Daniel; Zuzarte, Monica; Ribeiro Rodrigues, Teresa M.; Girao, Henrique; de Almeida, Luis Pereira ;
PUBLISHED: 2025, SOURCE: CELL DEATH & DISEASE, VOLUME: 16, ISSUE: 1
INDEXED IN: Scopus WOS CrossRef
3
TITLE: Differential impact of mutant Ataxin-3 in hindbrain regions: further evidence of white matter loss as a core pathological feature  Full Text
AUTHORS: Carina Henriques; Marta Silva; António Silva; David Rufino Ramos; Miguel Monteiro Lopes; Romina Aron Badin; Philippe Hantraye; Luís Pereira de Almeida ; Rui Jorge Nobre;
PUBLISHED: 2025, SOURCE: Experimental Neurology, VOLUME: 393
INDEXED IN: Scopus CrossRef: 1
4
TITLE: Exploring TLR agonists as adjuvants for COVID-19 oral vaccines  Full Text
AUTHORS: Felix, Paulo; Melo, Alexandra A.; Costa, Joao Panao; Colaco, Mariana; Pereira, Dina; Nunez, Jisette; de Almeida, Luis Pereira ; Borges, Olga;
PUBLISHED: 2025, SOURCE: VACCINE, VOLUME: 53
INDEXED IN: Scopus WOS CrossRef
5
TITLE: Extracellular vesicles-associated AAVs for the treatment of Machado-Joseph disease
AUTHORS: Carina Henriques; Patrícia Albuquerque; David Rufino-Ramos; Miguel M Lopes; Kevin Leandro; Catarina O Miranda; Rita Almeida; Guilherme Lopes-Gabriel; João de Sousa-Lourenço; Sara M Lopes; Laetitia S Gaspar; Diana D Lobo; Ana Carolina Silva; Teresa M Ribeiro-Rodrigues; Henrique Girão; Célia M Gomes; Rafael Baganha; Sónia Duarte; Casey A Maguire; Magda M Santana; Luís Pereira de Almeida ; Rui Jorge Nobre; ...More
PUBLISHED: 2025, SOURCE: Molecular Therapy
INDEXED IN: CrossRef
7
TITLE: Influence of ATXN2 intermediate CAG repeats, 9bp duplication and alternative splicing on SCA3 pathogenesis
AUTHORS: Lauerer, Marilena; Faber, Jennifer; Casadei, Nicolas; Santana, Magda M.; Auburger, Georg; Pogoda, Michaela; Admard, Jakob; Kaupp, Lea; Kos, Patricia Laura; Raposo, Mafalda; Lima, Manuela; de Almeida, Luis Pereira ; Garcia Moreno, Hector; Giunti, Paola; de Vries, Jeroen; van de Warrenburg, Bart P.; van Gaalen, Judith; Grobe Einsler, Marcus; Koyak, Berkan; Reetz, Kathrin; Erdlenbruch, Friedrich; Jacobi, Heike; Infante, Jon; Hengel, Holger; Schoels, Ludger; Klockgether, Thomas; Riess, Olaf; Huebener Schmid, Jeannette; ...More
PUBLISHED: 2025, SOURCE: ACTA NEUROPATHOLOGICA COMMUNICATIONS, VOLUME: 13, ISSUE: 1
INDEXED IN: Scopus WOS CrossRef
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TITLE: Progression of biological markers in spinocerebellar ataxia type 3: longitudinal analysis of prospective data from the ESMI cohort
AUTHORS: Berger, Moritz; Garcia Moreno, Hector; Ferreira, Monica; Hubener Schmid, Jeannette; Schaprian, Tamara; Wegner, Philipp; Elter, Tim; Teichmann, Kennet M.; Santana, Magda M.; Grobe Einsler, Marcus; Oender, Demet; Koyak, Berkan S. C.; Bernsen, Sarah; de Almeida, Luis Pereira ; Silva, Patrick; Ribeiro, Joana Afonso; Cunha, Lines; Gonzalez Robles, Cristina; Khan, Shamsher; Heslegrave, Amanda; Zetterberg, Henrik; Lima, Manuela; Raposo, Mafalda; Ferreira, Ana F.; Vasconcelos, Joao; van de Warrenburg, Bart P.; van Gaalen, Judith; van Prooije, Teije H.; de Vries, Eroen; Schols, Ludger; Riess, Olaf; Synofzik, Matthis; Timmann, Dagmar; Thieme, Andreas; Erdlenbruch, Friedrich; Infante, Jon; Pelayo Negro, Ana L.; Manrique, Leire; Reetz, Kathrin; Dogan, Imis; Oz, Gulin; Joers, JamesM.; Bushara, Khalafalla; Onyike, Chiadikaobi; Povazan, Michal; Jacobi, Heike; Schmahmann, Jeremy D.; Ratai, Eva Maria; Schmid, Matthias; Giunti, Paola; Klockgether, Thomas; Faber, Jennifer; ...More
PUBLISHED: 2025, SOURCE: LANCET REGIONAL HEALTH-EUROPE, VOLUME: 55
INDEXED IN: Scopus WOS CrossRef: 1
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TITLE: rAAV-Delivered Bicistronic Artificial microRNAs for Allele-Specific Silencing Improve Motor and Molecular Outcomes in Spinocerebellar Ataxia Type 3
AUTHORS: Ana Carolina Silva; Carina Henriques; Diana D Lobo; Ana Rita Fernandes; Miguel M Lopes; Kevin Leandro; Dina Pereira; Sónia P Duarte; Sara M Lopes; Magda M Santana; Amal Dakka; Steve De Marco; Marla Weetall; Jana Narasimhan; Anu Bhattacharyya; Rui Jorge Nobre; Luís Pereira de Almeida ;
PUBLISHED: 2025
INDEXED IN: CrossRef
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TITLE: Regional distribution of polymorphisms associated to the disease-causing gene of spinocerebellar ataxia type 3
AUTHORS: Tim Lukas Elter; Daniel Sturm; Magda Santana; Tamara Schaprian; Mafalda Raposo; Ana Rosa Vieira Melo; Manuela Lima ; Berkan Koyak; Demet Oender; Marcus Grobe Einsler; Sara Lopes; Patrick Silva; Luís Pereira de Almeida ; Paola Giunti; Hector Garcia Moreno; Suran Nethisinhe; Jeroen de Vries; Bart van de Warrenburg; Judith van Gaalen; Matthis Synofzik; Ludger Schöls; Kathrin Reetz; Friedrich Erdlenbruch; Heike Jacobi; Jon Infante; Olaf Riess; Thomas Klockgether; Andreas Thieme; Dagmar Timmann; Jennifer Faber; Jeannette Hübener Schmid; ...More
PUBLISHED: 2025, SOURCE: JOURNAL OF NEUROLOGY, VOLUME: 272, ISSUE: 1
INDEXED IN: Scopus WOS CrossRef
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