The Gene Therapy CoE at the Center of Portugal

Project Information

GeneT

Grant agreement ID: 101059981

DOI

10.3030/101059981

EC signature date 31 July 2023

Start date 1 January 2024

End date 31 December 2029

Funded under

• Widening participation and spreading excellence

Total cost

€ 14 003 705,00

EU contribution

€ 14 003 705,00

Coordinated by

UNIVERSIDADE DE COIMBRA
Portugal

CORDIS provides links to public deliverables and publications of HORIZON projects.

Links to deliverables and publications from FP7 projects, as well as links to some specific result types such as dataset and software, are dynamically retrieved from OpenAIRE .

Publications

Peer reviewed articles (24)

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

Author(s): 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
Published in: Acta Neuropathologica Communications, Issue 13, 2025, ISSN 2051-5960
Publisher: 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

Author(s): 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
Published in: Journal of Neurology, Issue 272, 2025, ISSN 0340-5354
Publisher: Springer Science and Business Media LLC
DOI: 10.1007/S00415-025-13024-0

Advanced Drug Delivery Reviews

Author(s): 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
Published in: ADVANCED DRUG DELIVERY REVIEWS, 2024, ISSN 0169-409X
Publisher: Elsevier BV
DOI: 10.1016/J.ADDR.2024.115346

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

Author(s): Margarida Vaz, Tânia Soares Martins, Kevin Leandro, Luís Pereira de Almeida, Odete A. B. da Cruz e Silva, Alexandra Nunes, Ana Gabriela Henriques
Published in: Molecules, Issue 30, 2025, ISSN 1420-3049
Publisher: MDPI AG
DOI: 10.3390/MOLECULES30020258

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

Author(s): 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
Published in: Journal of Extracellular Vesicles, Issue 13, 2024, ISSN 2001-3078
Publisher: Wiley
DOI: 10.1002/JEV2.12404

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

Author(s): Melvin A. Ambele, Catarina O. Miranda
Published in: Frontiers in Genetics, Issue 15, 2024, ISSN 1664-8021
Publisher: Frontiers Media SA
DOI: 10.3389/FGENE.2024.1439306

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

Author(s): 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
Published in: Advanced Drug Delivery Reviews, Issue 211, 2024, ISSN 0169-409X
Publisher: Elsevier BV
DOI: 10.1016/J.ADDR.2024.115346

Spinocerebellar ataxia type 3: from pathogenesis to promising therapeutics

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

Molecular therapy for polyQ disorders: from bench to clinical trials

Author(s): de Sousa-Lourenco, Joao; Silva, Ana C.; de Almeida, Luis Pereira; Nobre, Rui J.
Published in: TRENDS IN MOLECULAR MEDICINE, 2024, ISSN 1471-4914
Publisher: 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

Author(s): 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 in: Experimental Neurology, Issue 393, 2025, ISSN 0014-4886
Publisher: Elsevier BV
DOI: 10.1016/J.EXPNEUROL.2025.115413

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

Author(s): 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
Published in: Acta Neuropathologica Communications, Issue 13, 2025, ISSN 2051-5960
Publisher: Springer Science and Business Media LLC
DOI: 10.1186/S40478-025-02074-0

Cell Death and Disease

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

Viral-based animal models in polyglutamine disorders

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

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

Author(s): 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;
Published in: Brain, 2025, ISSN 0006-8950
Publisher: Oxford University Press
DOI: 10.1093/BRAIN/AWAF199

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

Author(s): Ricardo Moreira, Clévio Nóbrega, Luís Pereira de Almeida, Liliana Mendonça
Published in: Journal of Nanobiotechnology, Issue 22, 2024, ISSN 1477-3155
Publisher: 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

Author(s): Mariana Colaço, Maria T. Cruz, Luís Pereira de Almeida, Olga Borges
Published in: Pharmaceutics, Issue 16, 2025, ISSN 1999-4923
Publisher: MDPI AG
DOI: 10.3390/PHARMACEUTICS16101308

Exploring TLR agonists as adjuvants for COVID-19 oral vaccines

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

Journal of Neurology

Author(s): 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
Published in: Journal of Neurology, 2025, ISSN 0340-5354
Publisher: 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

Author(s): 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
Published in: JOURNAL OF VISUALIZED EXPERIMENTS, 2024, ISSN 1940-087X
Publisher: 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

Author(s): Liliana S. Mendonça, Ricardo Moreira, Daniel Henriques, Mónica Zuzarte, Teresa M. Ribeiro-Rodrigues, Henrique Girão, Luís Pereira de Almeida
Published in: Cell Death & Disease, Issue 16, 2025, ISSN 2041-4889
Publisher: 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

Author(s): 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
Published in: Neurobiology of Disease, Issue 193, 2024, ISSN 0969-9961
Publisher: Elsevier BV
DOI: 10.1016/J.NBD.2024.106456

Molecular therapy for polyQ disorders: from bench to clinical trials

Author(s): João de Sousa-Lourenço, Ana C. Silva, Luís Pereira de Almeida, Rui J. Nobre
Published in: Trends in Molecular Medicine, Issue 30, 2024, ISSN 1471-4914
Publisher: Elsevier BV
DOI: 10.1016/J.MOLMED.2024.05.004

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

Author(s): 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
Published in: International Journal of Biological Macromolecules, Issue 259, 2024, ISSN 0141-8130
Publisher: Elsevier BV
DOI: 10.1016/J.IJBIOMAC.2023.129157

Frontiers in Genetics

Author(s): Melvin A. Ambele; Melvin A. Ambele; Catarina O. Miranda; Catarina O. Miranda; Catarina O. Miranda; Catarina O. Miranda
Published in: Frontiers in Genetics, 2024, ISSN 1664-8021
Publisher: 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

Author(s): Colaço, Mariana Raquel Antunes
Published in: 2025
Publisher: University of Coimbra

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

Author(s): Saraiva, Sofia Teixeira
Published in: 2024
Publisher: University of Coimbra

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

Author(s): Barros, Maria Inês Veiga de Almeida
Published in: 2024
Publisher: Universidade de Coimbra