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TITLE: Extracellular vesicles-associated AAVs for the treatment of Machado-Joseph disease
AUTHORS: Henriques, Carina; Albuquerque, Patricia; Rufino Ramos, David; Lopes, Miguel M.; Leandro, Kevin; Miranda, Catarina O. ; Almeida, Rita; Lopes Gabriel, Guilherme; de Sousa Lourenco, Joao; Lopes, Sara M.; Gaspar, Laetitia S.; Lobo, Diana D.; Silva, Ana Carolina; Ribeiro Rodrigues, Teresa M.; Girao, Henrique; Gomes, Celia M.; Baganha, Rafael; Duarte, Sonia; Maguire, Casey A.; Santana, Magda M.; de Almeida, Luis Pereira ; Nobre, Rui Jorge; ...More
PUBLISHED: 2026, SOURCE: MOLECULAR THERAPY, VOLUME: 34, ISSUE: 1
INDEXED IN: Scopus WOS CrossRef
IN MY: ORCID
2
TITLE: A systematic screening assay identifies efficient small guide RNAs for CRISPR activation
AUTHORS: Arvidsson, Elin; Lobo, Diana Duarte; Sabarese, Ermelinda; Duarte, Fabio; Nobre, Rui Jorge; Quintino, Luis; Lundberg, Cecilia;
PUBLISHED: 2025, SOURCE: FRONTIERS IN BIOENGINEERING AND BIOTECHNOLOGY, VOLUME: 13
INDEXED IN: Scopus WOS CrossRef
IN MY: ORCID
3
TITLE: Gene Editing forATXN3Inactivation in Machado-Joseph disease: CRISPR-Cas9 as a Therapeutic Alternative to TALEN-Induced Toxicity
AUTHORS: Sara Monteiro Lopes; Miguel Monteiro Lopes; Daniela Oliveira; Magda Matos Santana; Ana Rita Fernandes; Ana Vasconcelos-Ferreira; Dina Pereira; Maria Casquinha; Clévio Nóbrega; Carlos A Matos; Neville E Sanjana; Patrick D Hsu; Fei Ann Ran; Lukasz Swiech; Le Cong; Feng Zhang; Rui Jorge Nobre; Luís Pereira de Almeida;
PUBLISHED: 2025
INDEXED IN: CrossRef
IN MY: ORCID
4
TITLE: A systematic screening assay identifies efficient small guide RNAs for CRISPR activation
AUTHORS: Elin Arvidsson; Diana Duarte Lobo; Ermelinda Sabarese; Fabio Duarte; Rui Jorge Nobre; Luis Quintino; Cecilia Lundberg;
PUBLISHED: 2025, SOURCE: Frontiers in Bioengineering and Biotechnology, VOLUME: 13
INDEXED IN: CrossRef
IN MY: ORCID
5
TITLE: Targeting under-screened women in cervical cancer: combining self-sampling and human papillomavirus testing with a strategic reminder plan
AUTHORS: Pereira, Sara da Graca; Nobre, Luis; Ribeiro, Marina; Carvalho, Patricia; Morais, Ana; Sousa, Rita; Moniz, Ana Paula; Matos, Francisco; Fernandes, Graca; Pimentel, Joao Pedro; Marinho, Jose Carlos; Sa, Jose Luis; Ilheu, Olga; Rebelo, Teresa; Fonseca Moutinho, Jose; Prazeres, Hugo; Nobre, Rui Jorge; Loureiro, Fernanda;
PUBLISHED: 2025, SOURCE: EUROPEAN JOURNAL OF PUBLIC HEALTH, VOLUME: 35, ISSUE: 5
INDEXED IN: Scopus WOS CrossRef
IN MY: ORCID
6
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
IN MY: ORCID
7
TITLE: Spinocerebellar ataxia type 3: from pathogenesis to promising therapeutics  Full Text
AUTHORS: Silva, Ana Carolina; Lopes, Miguel Monteiro; Lobo, Diana Duarte; Henriqu, Carina; de Almeida, Luis Pereira ; Nobre, Rui Jorge;
PUBLISHED: 2025, SOURCE: TRENDS IN MOLECULAR MEDICINE, VOLUME: 31, ISSUE: 11
INDEXED IN: Scopus WOS CrossRef
IN MY: ORCID
8
TITLE: Innate immunocompetent iNSpheroids: A hiPSC-derived 3D model to study the central nervous system captures an early CNS response to rAAV
AUTHORS: Catarina M Gomes; Gabriela Silva; Mafalda Aleixo; Daniel Simão; Stephan J Holtkamp; Diana D Lobo; Pradeep Harish; Rosalind Jenkins; Lek Dahal; Rui J Nobre; Luís P Almeida; Mark Trautwein; Paula M Alves; Catarina Brito;
PUBLISHED: 2025
INDEXED IN: CrossRef
IN MY: ORCID
9
TITLE: Astrocytic mGluR5 signaling tunes emotional and cognitive processing in the adult brain
AUTHORS: João Filipe Viana; José Duarte Dias; Candela González-Arias; Luís Samuel Alves; Alexandra Veiga; Daniela Sofia Abreu; João Luís Machado; Sara Barsanti; Rui Jorge Nobre; Luís Pereira de Almeida; Gertrudis Perea; João Filipe Oliveira;
PUBLISHED: 2025
INDEXED IN: CrossRef
IN MY: ORCID
10
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
IN MY: ORCID
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