The Gene Therapy CoE at the Center of Portugal

Informazioni relative al progetto

GeneT

ID dell’accordo di sovvenzione: 101059981

DOI

10.3030/101059981

Data della firma CE 31 Luglio 2023

Data di avvio 1 Gennaio 2024

Data di completamento 31 Dicembre 2029

Finanziato da

• Widening participation and spreading excellence

Costo totale

€ 14 003 705,00

Contributo UE

€ 14 003 705,00

Coordinato da

UNIVERSIDADE DE COIMBRA
Portugal

CORDIS fornisce collegamenti ai risultati finali pubblici e alle pubblicazioni dei progetti ORIZZONTE.

I link ai risultati e alle pubblicazioni dei progetti del 7° PQ, così come i link ad alcuni tipi di risultati specifici come dataset e software, sono recuperati dinamicamente da .OpenAIRE .

Pubblicazioni

Peer reviewed articles (24)

Influence of ATXN2 intermediate CAG repeats, 9bp duplication and alternative splicing on SCA3 pathogenesis

Autori: Marilena Lauerer, Jennifer Faber, Nicolas Casadei, Magda M. Santana, Georg Auburger, Michaela Pogoda, Jakob Admard, Lea Kaupp, Patricia Laura Kos, Mafalda Raposo, Manuela Lima, Luis Pereira de Almeida, Hector Garcia-Moreno, Paola Giunti, Jeroen de Vries, Bart P. van de Warrenburg, Judith van Gaalen, Marcus Grobe-Einsler, Berkan Koyak, Kathrin Reetz, Friedrich Erdlenbruch, Heike Jacobi, Jon Infante, Holger Hengel, Ludger Schöls, Thomas Klockgether, Olaf Rieß, Jeannette Hübener-Schmid, null null
Pubblicato in: Acta Neuropathologica Communications, Numero 13, 2025, ISSN 2051-5960
Editore: Springer Science and Business Media LLC
DOI: 10.1186/S40478-025-02074-0

Longitudinal description of health-related quality of life and depressive symptoms in polyQ spinocerebellar ataxia patients

Autori: Audrey Iskandar, Maresa Buchholz, Iris Blotenberg, Tanja Schmitz-Hübsch, Jennifer Faber, Heike Jacobi, Feng Xie, Marcus Grobe-Einsler, Thomas Klockgether, Bernhard Michalowsky, null null, Kathrin Reetz, Mafalda Raponso, Carlos Gonzales, Berkan Koyak, Demet Oender, Luís Pereira de Almeida, Patrick Silva, Joana Afonso Ribeiro, Andreas Thieme, Friedrich Erdlenbruch, Jeroen de Vries, Chiadikaobi Onyike, Paola Giunti, Hector Garcia-Moreno, Gülin Öz, Almut Turid Bischoff, Bart van de Warrenburg, Judith van Gaalen, Jon Infante, Leire Manrique, Ludger Schöls, Olaf Riess, Sophie Tezenas du Montcel, Peter Bauer, Paola Giunti, Arron Cook, Robyn Labrum, Michael H. Parkinson, Alexandra Durr, Alexis Brice, Perrine Charles, Cecilia Marelli, Caterina Mariotti, Lorenzo Nanetti, Marta Panzeri, Maria Rakowicz, Anna Sulek, Anna Sobanska, Holger Hengel, Laszlo Baliko, Bela Melegh, Alessandro Filla, Antonella Antenora, José Berciano, Dagmar Timmann, Sandra Szymanski, Sylvia Boesch, Jun-Suk Kang, Massimo Padolfo, Jörg B. Schulz, Sonia Molho, Alhassane Diallo
Pubblicato in: Journal of Neurology, Numero 272, 2025, ISSN 0340-5354
Editore: Springer Science and Business Media LLC
DOI: 10.1007/S00415-025-13024-0

Advanced Drug Delivery Reviews

Autori: Leandro, Kevin; Rufino-Ramos, David; Breyne, Koen; Di Ianni, Emilio; Lopes, Sara M.; Nobre, Rui Jorge; Kleinstiver, Benjamin P.; Perdiga, Pedro R. L.; Breakefield, Xandra O.; de Almeida, Luis Pereira
Pubblicato in: ADVANCED DRUG DELIVERY REVIEWS, 2024, ISSN 0169-409X
Editore: Elsevier BV
DOI: 10.1016/J.ADDR.2024.115346

Fourier Transform Infrared Spectroscopy Analysis as a Tool to Address Aβ Impact on Extracellular Vesicles

Autori: Margarida Vaz, Tânia Soares Martins, Kevin Leandro, Luís Pereira de Almeida, Odete A. B. da Cruz e Silva, Alexandra Nunes, Ana Gabriela Henriques
Pubblicato in: Molecules, Numero 30, 2025, ISSN 1420-3049
Editore: MDPI AG
DOI: 10.3390/MOLECULES30020258

Minimal information for studies of extracellular vesicles (MISEV2023): From basic to advanced approaches

Autori: Joshua A. Welsh, Deborah C. I. Goberdhan, Lorraine O'Driscoll, Edit I. Buzas, Cherie Blenkiron, Benedetta Bussolati, Houjian Cai, Dolores Di Vizio, Tom A. P. Driedonks, Uta Erdbrügger, Juan M. Falcon‐Perez, Qing‐Ling Fu, Andrew F. Hill, Metka Lenassi, Sai Kiang Lim, Mỹ G. Mahoney, Sujata Mohanty, Andreas Möller, Rienk Nieuwland, Takahiro Ochiya, Susmita Sahoo, Ana C. Torrecilhas, Lei Zheng, Andries Zijlstra, Sarah Abuelreich, Reem Bagabas, Paolo Bergese, Esther M. Bridges, Marco Brucale, Dylan Burger, Randy P. Carney, Emanuele Cocucci, Rossella Crescitelli, Edveena Hanser, Adrian L. Harris, Norman J. Haughey, An Hendrix, Alexander R. Ivanov, Tijana Jovanovic‐Talisman, Nicole A. Kruh‐Garcia, Vroniqa Ku'ulei‐Lyn Faustino, Diego Kyburz, Cecilia Lässer, Kathleen M. Lennon, Jan Lötvall, Adam L. Maddox, Elena S. Martens‐Uzunova, Rachel R. Mizenko, Lauren A. Newman, Andrea Ridolfi, Eva Rohde, Tatu Rojalin, Andrew Rowland, Andras Saftics, Ursula S. Sandau, Julie A. Saugstad, Faezeh Shekari, Simon Swift, Dmitry Ter‐Ovanesyan, Juan P. Tosar, Zivile Useckaite, Francesco Valle, Zoltan Varga, Edwin van der Pol, Martijn J. C. van Herwijnen, Marca H. M. Wauben, Ann M. Wehman, Sarah Williams, Andrea Zendrini, Alan J. Zimmerman, null null, Clotilde Théry, Kenneth W. Witwer
Pubblicato in: Journal of Extracellular Vesicles, Numero 13, 2024, ISSN 2001-3078
Editore: Wiley
DOI: 10.1002/JEV2.12404

Editorial: Mesenchymal and induced-pluripotent stem cells as models to study biological processes

Autori: Melvin A. Ambele, Catarina O. Miranda
Pubblicato in: Frontiers in Genetics, Numero 15, 2024, ISSN 1664-8021
Editore: Frontiers Media SA
DOI: 10.3389/FGENE.2024.1439306

Exploring the potential of cell-derived vesicles for transient delivery of gene editing payloads

Autori: Kevin Leandro, David Rufino-Ramos, Koen Breyne, Emilio Di Ianni, Sara M. Lopes, Rui Jorge Nobre, Benjamin P. Kleinstiver, Pedro R.L. Perdigão, Xandra O. Breakefield, Luís Pereira de Almeida
Pubblicato in: Advanced Drug Delivery Reviews, Numero 211, 2024, ISSN 0169-409X
Editore: Elsevier BV
DOI: 10.1016/J.ADDR.2024.115346

Spinocerebellar ataxia type 3: from pathogenesis to promising therapeutics

Autori: Ana Carolina Silva, Miguel Monteiro Lopes, Diana Duarte Lobo, Carina Henriques, Luís Pereira de Almeida, Rui Jorge Nobre
Pubblicato in: Trends in Molecular Medicine, 2025, ISSN 1471-4914
Editore: Elsevier BV
DOI: 10.1016/J.MOLMED.2025.06.007

Molecular therapy for polyQ disorders: from bench to clinical trials

Autori: de Sousa-Lourenco, Joao; Silva, Ana C.; de Almeida, Luis Pereira; Nobre, Rui J.
Pubblicato in: TRENDS IN MOLECULAR MEDICINE, 2024, ISSN 1471-4914
Editore: CELL PRESS
DOI: 10.1016/J.MOLMED.2024.05.004

Differential impact of mutant Ataxin-3 in hindbrain regions: further evidence of white matter loss as a core pathological feature

Autori: Carina Henriques, Marta Silva, António Silva, David Rufino-Ramos, Miguel Monteiro Lopes, Romina Aron Badin, Philippe Hantraye, Luís Pereira de Almeida, Rui Jorge Nobre
Pubblicato in: Experimental Neurology, Numero 393, 2025, ISSN 0014-4886
Editore: Elsevier BV
DOI: 10.1016/J.EXPNEUROL.2025.115413

Influence of ATXN2 intermediate CAG repeats, 9bp duplication and alternative splicing on SCA3 pathogenesis

Autori: Marilena Lauerer, Jennifer Faber, Nicolas Casadei, Magda M. Santana, Georg Auburger, Michaela Pogoda, Jakob Admard, Lea Kaupp, Patricia Laura Kos, Mafalda Raposo, Manuela Lima, Luis Pereira de Almeida, Hector Garcia-Moreno, Paola Giunti, Jeroen de Vries, Bart P. van de Warrenburg, Judith van Gaalen, Marcus Grobe-Einsler, Berkan Koyak, Kathrin Reetz, Friedrich Erdlenbruch, Heike Jacobi, Jon Infante, Holger Hengel, Ludger Schöls, Thomas Klockgether, Olaf Rieß, Jeannette Hübener-Schmid, null null
Pubblicato in: Acta Neuropathologica Communications, Numero 13, 2025, ISSN 2051-5960
Editore: Springer Science and Business Media LLC
DOI: 10.1186/S40478-025-02074-0

Cell Death and Disease

Autori: Liliana S. Mendonça; Ricardo Moreira; Daniel Henriques; Mónica Zuzarte; Teresa M. Ribeiro-Rodrigues; Henrique Girão; Luís Pereira de Almeida
Pubblicato in: Cell Death & Disease, 2025, ISSN 2041-4889
Editore: Nature Publishing Group
DOI: 10.1038/S41419-025-07659-0

Viral-based animal models in polyglutamine disorders

Autori: Carina Henriques, Miguel M Lopes, Ana C Silva, Diana D Lobo, Romina Aron Badin, Philippe Hantraye, Luís Pereira de Almeida, Rui Jorge Nobre
Pubblicato in: Brain, Numero 147, 2024, ISSN 0006-8950
Editore: Oxford University Press (OUP)
DOI: 10.1093/BRAIN/AWAE012

Circadian rhythms are disrupted in patients and preclinical models of Machado-Joseph disease

Autori: Rodrigo F N Ribeiro; Dina Pereira; Sara M Lopes; Tiago Reis; Patrick Silva; Diana D Lobo; Laetitia S Gaspar; João Durães; Ana Rita Fernandes; Marisa Ferreira-Marques; Catarina Carvalhas-Almeida; João Peça; Ana Rita Álvaro; Isabel Santana; Magda M Santana;
Pubblicato in: Brain, 2025, ISSN 0006-8950
Editore: Oxford University Press
DOI: 10.1093/BRAIN/AWAF199

Brain-targeted drug delivery - nanovesicles directed to specific brain cells by brain-targeting ligands

Autori: Ricardo Moreira, Clévio Nóbrega, Luís Pereira de Almeida, Liliana Mendonça
Pubblicato in: Journal of Nanobiotechnology, Numero 22, 2024, ISSN 1477-3155
Editore: Springer Science and Business Media LLC
DOI: 10.1186/S12951-024-02511-7

Mannose and Lactobionic Acid in Nasal Vaccination: Enhancing Antigen Delivery via C-Type Lectin Receptors

Autori: Mariana Colaço, Maria T. Cruz, Luís Pereira de Almeida, Olga Borges
Pubblicato in: Pharmaceutics, Numero 16, 2025, ISSN 1999-4923
Editore: MDPI AG
DOI: 10.3390/PHARMACEUTICS16101308

Exploring TLR agonists as adjuvants for COVID-19 oral vaccines

Autori: Felix, Paulo; Melo, Alexandra A.; Costa, Joao Panao; Colaco, Mariana; Pereira, Dina; Nunez, Jisette; de Almeida, Luis Pereira; Borges, Olga
Pubblicato in: Vaccine, 2025, ISSN 0264-410X
Editore: ELSEVIER SCI LTD
DOI: 10.1016/J.VACCINE.2025.127078

Journal of Neurology

Autori: Audrey Iskandar; Maresa Buchholz; Iris Blotenberg; Tanja Schmitz-Hübsch; Jennifer Faber; Heike Jacobi; Feng Xie; Marcus Grobe-Einsler; Thomas Klockgether; Bernhard Michalowsky; Kathrin Reetz; Mafalda Raponso; Carlos Gonzales; Berkan Koyak; Demet Oender; Luís Pereira de Almeida; Patrick Silva; Joana Afonso Ribeiro; Andreas Thieme; Friedrich Erdlenbruch; Jeroen de Vries; Chiadikaobi Onyike; Paola Giunti; Hector Garcia-Moreno; Gülin Öz; Almut Turid Bischoff; Bart van de Warrenburg; Judith van Gaalen; Jon Infante; Leire Manrique; Ludger Schöls; Olaf Riess; Sophie Tezenas du Montcel; Peter Bauer; Paola Giunti; Arron Cook; Robyn Labrum; Michael H. Parkinson; Alexandra Durr; Alexis Brice; Perrine Charles; Cecilia Marelli; Caterina Mariotti; Lorenzo Nanetti; Marta Panzeri; Maria Rakowicz; Anna Sulek; Anna Sobanska; Holger Hengel; Laszlo Baliko; Bela Melegh; Alessandro Filla; Antonella Antenora; José Berciano; Dagmar Timmann; Sandra Szymanski; Sylvia Boesch; Jun-Suk Kang; Massimo Padolfo; Jörg B. Schulz; Sonia Molho; Alhassane Diallo
Pubblicato in: Journal of Neurology, 2025, ISSN 0340-5354
Editore: Dr. Dietrich Steinkopff Verlag
DOI: 10.1007/S00415-025-13024-0

Isolation of Adeno-associated Viral Vectors Through a Single-step and Semi-automated Heparin Affinity Chromatography Protocol

Autori: Lopes, Miguel M.; Lopes, Sara M.; Baganha, Rafael; Henriques, Carina; Silva, Ana C.; Lobo, Diana D.; Cortes, Luisa; de Almeida, Luis Pereira; Nobre, Rui Jorge
Pubblicato in: JOURNAL OF VISUALIZED EXPERIMENTS, 2024, ISSN 1940-087X
Editore: JOURNAL OF VISUALIZED EXPERIMENTS
DOI: 10.3791/66550

Autophagy- and oxidative stress-related protein deregulation mediated by extracellular vesicles of human MJD/SCA3 iPSC-derived neuroepithelial stem cells and differentiated neural cultures

Autori: Liliana S. Mendonça, Ricardo Moreira, Daniel Henriques, Mónica Zuzarte, Teresa M. Ribeiro-Rodrigues, Henrique Girão, Luís Pereira de Almeida
Pubblicato in: Cell Death & Disease, Numero 16, 2025, ISSN 2041-4889
Editore: Springer Science and Business Media LLC
DOI: 10.1038/S41419-025-07659-0

Blood and cerebellar abundance of ATXN3 splice variants in spinocerebellar ataxia type 3/Machado-Joseph disease

Autori: Mafalda Raposo, Jeannette Hübener-Schmid, Rebecca Tagett, Ana F. Ferreira, Ana Rosa Vieira Melo, João Vasconcelos, Paula Pires, Teresa Kay, Hector Garcia-Moreno, Paola Giunti, Magda M. Santana, Luis Pereira de Almeida, Jon Infante, Bart P. van de Warrenburg, Jeroen J. de Vries, Jennifer Faber, Thomas Klockgether, Nicolas Casadei, Jakob Admard, Ludger Schöls, Olaf Riess, Maria do Carmo Costa, Manuela Lima
Pubblicato in: Neurobiology of Disease, Numero 193, 2024, ISSN 0969-9961
Editore: Elsevier BV
DOI: 10.1016/J.NBD.2024.106456

Molecular therapy for polyQ disorders: from bench to clinical trials

Autori: João de Sousa-Lourenço, Ana C. Silva, Luís Pereira de Almeida, Rui J. Nobre
Pubblicato in: Trends in Molecular Medicine, Numero 30, 2024, ISSN 1471-4914
Editore: Elsevier BV
DOI: 10.1016/J.MOLMED.2024.05.004

RNA-based liposomes for oral cancer: From biophysical characterization to biological evaluation

Autori: Jéssica Lopes-Nunes, Pedro Simões, David Moreira, Kevin Leandro, Rui Jorge Nobre, Luís Pereira de Almeida, Maria Paula Cabral Campello, Maria Cristina Oliveira, António Paulo, Ana Coutinho, Ana M. Melo, Cândida Tomaz, Carla Cruz
Pubblicato in: International Journal of Biological Macromolecules, Numero 259, 2024, ISSN 0141-8130
Editore: Elsevier BV
DOI: 10.1016/J.IJBIOMAC.2023.129157

Frontiers in Genetics

Autori: Melvin A. Ambele; Melvin A. Ambele; Catarina O. Miranda; Catarina O. Miranda; Catarina O. Miranda; Catarina O. Miranda
Pubblicato in: Frontiers in Genetics, 2024, ISSN 1664-8021
Editore: FRONTIERS MEDIA SA
DOI: 10.3389/FGENE.2024.1439306

Thesis and dissertations (3)

Design of Functionalized Chitosan-DNA Complexes and Nanoparticles for Nasal Immunization against SARS-CoV-2

Autori: Colaço, Mariana Raquel Antunes
Pubblicato in: 2025
Editore: University of Coimbra

Development of polymeric membranes for the prevention of post-operative tendon adhesions

Autori: Saraiva, Sofia Teixeira
Pubblicato in: 2024
Editore: University of Coimbra

Unraveling Neurovascular Dysfunctions in Machado-Joseph Disease: Mesenchymal Stromal Cells as a Promising Therapy

Autori: Barros, Maria Inês Veiga de Almeida
Pubblicato in: 2024
Editore: Universidade de Coimbra