The Gene Therapy CoE at the Center of Portugal

Información del proyecto

GeneT

Identificador del acuerdo de subvención: 101059981

DOI

10.3030/101059981

Fecha de la firma de la CE 31 Julio 2023

Fecha de inicio 1 Enero 2024

Fecha de finalización 31 Diciembre 2029

Financiado con arreglo a

• Widening participation and spreading excellence

Coste total

€ 14 003 705,00

Aportación de la UE

€ 14 003 705,00

Coordinado por

UNIVERSIDADE DE COIMBRA
Portugal

CORDIS proporciona enlaces a los documentos públicos y las publicaciones de los proyectos de los programas marco HORIZONTE.

Los enlaces a los documentos y las publicaciones de los proyectos del Séptimo Programa Marco, así como los enlaces a algunos tipos de resultados específicos, como conjuntos de datos y «software», se obtienen dinámicamente de OpenAIRE .

Publicaciones

Peer reviewed articles (24)

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

Autores: 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
Publicado en: Acta Neuropathologica Communications, Edición 13, 2025, ISSN 2051-5960
Editor: 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

Autores: 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
Publicado en: Journal of Neurology, Edición 272, 2025, ISSN 0340-5354
Editor: Springer Science and Business Media LLC
DOI: 10.1007/S00415-025-13024-0

Advanced Drug Delivery Reviews

Autores: 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
Publicado en: ADVANCED DRUG DELIVERY REVIEWS, 2024, ISSN 0169-409X
Editor: Elsevier BV
DOI: 10.1016/J.ADDR.2024.115346

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

Autores: Margarida Vaz, Tânia Soares Martins, Kevin Leandro, Luís Pereira de Almeida, Odete A. B. da Cruz e Silva, Alexandra Nunes, Ana Gabriela Henriques
Publicado en: Molecules, Edición 30, 2025, ISSN 1420-3049
Editor: MDPI AG
DOI: 10.3390/MOLECULES30020258

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

Autores: 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
Publicado en: Journal of Extracellular Vesicles, Edición 13, 2024, ISSN 2001-3078
Editor: Wiley
DOI: 10.1002/JEV2.12404

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

Autores: Melvin A. Ambele, Catarina O. Miranda
Publicado en: Frontiers in Genetics, Edición 15, 2024, ISSN 1664-8021
Editor: Frontiers Media SA
DOI: 10.3389/FGENE.2024.1439306

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

Autores: 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
Publicado en: Advanced Drug Delivery Reviews, Edición 211, 2024, ISSN 0169-409X
Editor: Elsevier BV
DOI: 10.1016/J.ADDR.2024.115346

Spinocerebellar ataxia type 3: from pathogenesis to promising therapeutics

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

Molecular therapy for polyQ disorders: from bench to clinical trials

Autores: de Sousa-Lourenco, Joao; Silva, Ana C.; de Almeida, Luis Pereira; Nobre, Rui J.
Publicado en: TRENDS IN MOLECULAR MEDICINE, 2024, ISSN 1471-4914
Editor: 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

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

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

Autores: 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
Publicado en: Acta Neuropathologica Communications, Edición 13, 2025, ISSN 2051-5960
Editor: Springer Science and Business Media LLC
DOI: 10.1186/S40478-025-02074-0

Cell Death and Disease

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

Viral-based animal models in polyglutamine disorders

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

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

Autores: 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;
Publicado en: Brain, 2025, ISSN 0006-8950
Editor: Oxford University Press
DOI: 10.1093/BRAIN/AWAF199

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

Autores: Ricardo Moreira, Clévio Nóbrega, Luís Pereira de Almeida, Liliana Mendonça
Publicado en: Journal of Nanobiotechnology, Edición 22, 2024, ISSN 1477-3155
Editor: 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

Autores: Mariana Colaço, Maria T. Cruz, Luís Pereira de Almeida, Olga Borges
Publicado en: Pharmaceutics, Edición 16, 2025, ISSN 1999-4923
Editor: MDPI AG
DOI: 10.3390/PHARMACEUTICS16101308

Exploring TLR agonists as adjuvants for COVID-19 oral vaccines

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

Journal of Neurology

Autores: 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
Publicado en: Journal of Neurology, 2025, ISSN 0340-5354
Editor: 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

Autores: 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
Publicado en: JOURNAL OF VISUALIZED EXPERIMENTS, 2024, ISSN 1940-087X
Editor: 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

Autores: Liliana S. Mendonça, Ricardo Moreira, Daniel Henriques, Mónica Zuzarte, Teresa M. Ribeiro-Rodrigues, Henrique Girão, Luís Pereira de Almeida
Publicado en: Cell Death & Disease, Edición 16, 2025, ISSN 2041-4889
Editor: 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

Autores: 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
Publicado en: Neurobiology of Disease, Edición 193, 2024, ISSN 0969-9961
Editor: Elsevier BV
DOI: 10.1016/J.NBD.2024.106456

Molecular therapy for polyQ disorders: from bench to clinical trials

Autores: João de Sousa-Lourenço, Ana C. Silva, Luís Pereira de Almeida, Rui J. Nobre
Publicado en: Trends in Molecular Medicine, Edición 30, 2024, ISSN 1471-4914
Editor: Elsevier BV
DOI: 10.1016/J.MOLMED.2024.05.004

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

Autores: 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
Publicado en: International Journal of Biological Macromolecules, Edición 259, 2024, ISSN 0141-8130
Editor: Elsevier BV
DOI: 10.1016/J.IJBIOMAC.2023.129157

Frontiers in Genetics

Autores: Melvin A. Ambele; Melvin A. Ambele; Catarina O. Miranda; Catarina O. Miranda; Catarina O. Miranda; Catarina O. Miranda
Publicado en: Frontiers in Genetics, 2024, ISSN 1664-8021
Editor: 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

Autores: Colaço, Mariana Raquel Antunes
Publicado en: 2025
Editor: University of Coimbra

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

Autores: Saraiva, Sofia Teixeira
Publicado en: 2024
Editor: University of Coimbra

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

Autores: Barros, Maria Inês Veiga de Almeida
Publicado en: 2024
Editor: Universidade de Coimbra