Terapia Génica
Vetores Virais
Células Estaminais
Doenças Neurodegenerativas
Doença de Machado-Joseph
Modelos de doença
Mecanismos terapêuticos
Transplantação de células estaminais
Silenciamento genético
Investigação de biomarcadores de doença
O grupo dedica-se ao desenvolvimento de novas terapias translacionais para doenças cerebrais. Para tal, o grupo desenha e utiliza plataformas tecnológicas baseadas em vectores de transferência de genes virais e não virais e células estaminais pluripotentes induzidas para o:
O grupo resulta de uma reestruturação do antigo grupo “Vectores e Terapia Génica” e possui uma forte especialização em transferência de genes (virais e não virais) e células estaminais para modelização e terapia de doenças cerebrais. As nossas plataformas tecnológicas incluem:
Informação sobre artigos em revista, actualizada a 01-12-2024, a partir da plataforma CIÊNCIAVITAE.
Exploring the potential of cell-derived vesicles for transient delivery of gene editing payloads
Leandro, K.; Rufino-Ramos, D.; Breyne, K.; Di Ianni, E.; Lopes, S.M.; Jorge Nobre, R.; Kleinstiver, B.P.; et al, 2024. Advanced Drug Delivery Reviews. 211. 2024. http://www.scopus.com/inward/record.url?eid=2-s2.0-85197258413&partnerID=MN8TOARS . 10.1016/j.addr.2024.115346 . Advanced Drug Delivery Reviews
RNA-based liposomes for oral cancer: From biophysical characterization to biological evaluation
Lopes-Nunes, Jéssica; Simões, Pedro; Moreira, David; Leandro, Kevin; Nobre, Rui Jorge; Pereira de Almeida, Luís; Campello, Maria Paula Cabral; et al, 2024. International Journal of Biological Macromolecules. 259. 2024. http://dx.doi.org/10.1016/j.ijbiomac.2023.129157 . 10.1016/j.ijbiomac.2023.129157 . International Journal of Biological Macromolecules
Factors Influencing Health-Related Quality of Life of Patients with Spinocerebellar Ataxia
Weber, Niklas; Buchholz, Maresa; Rädke, Anika; Faber, Jennifer; Schmitz-Hübsch, Tanja; Jacobi, Heike; Klockgether, Thomas; et al, 2024. The Cerebellum. 2024. http://dx.doi.org/10.1007/s12311-024-01657-2 . 10.1007/s12311-024-01657-2 . The Cerebellum
Viral-based animal models in polyglutamine disorders
Henriques, Carina; Lopes, Miguel M; Silva, Ana C; Lobo, Diana D; Aron Badin, Romina; Hantraye, Philippe; Pereira de Almeida, Luís; Nobre, Rui Jorge, 2024. Brain. 2024. http://dx.doi.org/10.1093/brain/awae012 . 10.1093/brain/awae012 . Brain
Graft-derived neurons and bystander effects are maintained for six months after human iPSC-derived NESC transplantation in mice’s cerebella
Mendonça, Liliana S.; Henriques, Daniel; Fernandes, Vanessa; Moreira, Ricardo; Brás, João; Duarte, Sónia; Schwamborn, Jens C.; de Almeida, Luís Pereira, 2024. Scientific Reports. 1. 14. 2024. http://dx.doi.org/10.1038/s41598-024-53542-x . 10.1038/s41598-024-53542-x . Scientific Reports
Phosphoproteome Microarray Analysis of Extracellular Particles as a Tool to Explore Novel Biomarker Candidates for Alzheimer’s Disease
Soares Martins, Tânia; Pelech, Steven; Ferreira, Maria; Pinho, Beatriz; Leandro, Kevin; de Almeida, Luís Pereira; Breitling, Benedict; et al, 2024. International Journal of Molecular Sciences. 3. 25. 2024. http://dx.doi.org/10.3390/ijms25031584 . 10.3390/ijms25031584 . International Journal of Molecular Sciences
Isolation of Adeno-associated Viral Vectors Through a Single-step and Semi-automated Heparin Affinity Chromatography Protocol
Nobre, Rui Jorge; Pereira de Almeida, Luis Fernando Morgado, 2024. JoVE Journal. e66550. 2024. https://www.jove.com/t/66550 . 10.3791/66550 . in press JoVE Journal
Minimal information for studies of extracellular vesicles (MISEV2023): From basic to advanced approaches
Welsh, J.A.; Goberdhan, D.C.I.; O’Driscoll, L.; Buzas, E.I.; Blenkiron, C.; Bussolati, B.; Cai, H.; et al, 2024. Journal of Extracellular Vesicles. 2. 13. 2024. http://www.scopus.com/inward/record.url?eid=2-s2.0-85183359756&partnerID=MN8TOARS . 10.1002/jev2.12404 . Journal of Extracellular Vesicles
Molecular therapy for polyQ disorders: from bench to clinical trials
de Sousa-Lourenço, J.; Silva, A.C.; Pereira de Almeida, L.; Nobre, R.J., 2024. Trends in Molecular Medicine. 2024. http://www.scopus.com/inward/record.url?eid=2-s2.0-85195031116&partnerID=MN8TOARS . 10.1016/j.molmed.2024.05.004 . Trends in Molecular Medicine
Modeling Lysosomal Storage Disorders in an Innovative Way: Establishment and Characterization of Stem Cell Lines from Human Exfoliated Deciduous Teeth of Mucopolysaccharidosis Type II Patients
Carvalho, S.; Santos, J.I.; Moreira, L.; Duarte, A.J.; Gaspar, P.; Rocha, H.; Encarnação, M.; et al, 2024. International Journal of Molecular Sciences. 6. 25. 2024. http://www.scopus.com/inward/record.url?eid=2-s2.0-85189031362&partnerID=MN8TOARS . 10.3390/ijms25063546 . International Journal of Molecular Sciences
Brain-targeted drug delivery - nanovesicles directed to specific brain cells by brain-targeting ligands
Moreira, R.; Nóbrega, C.; de Almeida, L.P.; Mendonça, L., 2024. Journal of Nanobiotechnology. 1. 22. 2024. http://www.scopus.com/inward/record.url?eid=2-s2.0-85193498717&partnerID=MN8TOARS . 10.1186/s12951-024-02511-7 . Journal of Nanobiotechnology
Consensus Recommendations for Clinical Outcome Assessments and Registry Development in Ataxias: Ataxia Global Initiative (AGI) Working Group Expert Guidance
Klockgether, T.; Synofzik, M.; Alhusaini, S.; Anheim, M.; Antonijevic, I.; Ashizawa, T.; Bataller, L.; et al, 2023. Cerebellum. 2023. http://www.scopus.com/inward/record.url?eid=2-s2.0-85151668891&partnerID=MN8TOARS . 10.1007/s12311-023-01547-z . Cerebellum
The frequency of non-motor symptoms in SCA3 and their association with disease severity and lifestyle factors
Hengel, H.; Martus, P.; Faber, J.; Giunit, P.; Garcia-Moreno, H.; Solanky, N.; Klockgether, T.; et al, 2023. Journal of Neurology. 944 - 952. 2. 270. 2023. http://www.scopus.com/inward/record.url?eid=2-s2.0-85141199384&partnerID=MN8TOARS . 10.1007/s00415-022-11441-z . Journal of Neurology
Ataxin-2 in the hypothalamus at the crossroads between metabolism and clock genes
Carmo-Silva, S.; Ferreira-Marques, M.; Nóbrega, C.; Botelho, M.; Costa, D.; Aveleira, C.A.; Pulst, S.M.; et al, 2023. Journal of Molecular Endocrinology. 1. 70. 2023. http://www.scopus.com/inward/record.url?eid=2-s2.0-85143645604&partnerID=MN8TOARS . 10.1530/JME-21-0272 . under revision Journal of Molecular Endocrinology
A Novel Genetic Variant in MBD5 Associated with Severe Epilepsy and Intellectual Disability: Potential Implications on Neural Primary Cilia
Mariana Martins; Ana Rafaela Oliveira; Solange Martins; José Pedro Vieira; Pedro Perdigão; Ana Rita Fernandes; Luís Pereira de Almeida; et al, 2023. International Journal of Molecular Sciences. 2023. https://doi.org/10.3390/ijms241612603 . 10.3390/ijms241612603 . International Journal of Molecular Sciences
Isolation of Extracellular Vesicles from Human Follicular Fluid: Size-Exclusion Chromatography versus Ultracentrifugation
Maria Soares; Maria M. Pinto; Rui Jorge Nobre; Luís Pereira de Almeida; Maria da Graça Rasteiro; Teresa Almeida-Santos; João Ramalho-Santos; Ana Paula Sousa, 2023. Biomolecules. 2023. https://doi.org/10.3390/biom13020278 . 10.3390/biom13020278 . Biomolecules
Selective, broad-spectrum antiviral photodynamic disinfection with dicationic imidazolyl chlorin photosensitizers
Arnaut, Z.A.; Pinto, S.M.A.; Aroso, R.T.; Amorim, A.S.; Lobo, C.S.; Schaberle, F.A.; Pereira, D.; et al, 2023. Photochemical and Photobiological Sciences. 2023. http://www.scopus.com/inward/record.url?eid=2-s2.0-85172998540&partnerID=MN8TOARS . 10.1007/s43630-023-00476-4 . Photochemical and Photobiological Sciences
Blood transcriptome sequencing identifies biomarkers able to track disease stages in spinocerebellar ataxia type 3
Raposo, M.; Hübener-Schmid, J.; Ferreira, A.F.; Vieira Melo, A.R.; Vasconcelos, J.; Pires, P.; Kay, T.; et al, 2023. Brain. 4132 - 4143. 10. 146. 2023. http://www.scopus.com/inward/record.url?eid=2-s2.0-85173781207&partnerID=MN8TOARS . 10.1093/brain/awad128 . Brain
Extracellular communication between brain cells through functional transfer of Cre mRNA
Rufino-Ramos, David; Leandro, K; Perdigão, Pedro; O'Brien, K; Pinto, MM; Santana, MM; van Solinge, TS; et al, 2023. Molecular Therapy. 2023. http://www.scopus.com/inward/record.url?eid=2-s2.0-85160280091&partnerID=MN8TOARS . 10.1016/j.ymthe.2023.05.012 . in press Molecular Therapy
In Vitro Evaluation of Biphasic Calcium Phosphate Scaffolds Derived from Cuttlefish Bone Coated with Poly(ester urea) for Bone Tissue Regeneration
Patrícia Pereira; Ana S. Neto; Ana S. Rodrigues; Inês Barros; Catarina Miranda; João Ramalho-Santos; Luís Pereira de Almeida; et al, 2023. Polymers. 2023. https://doi.org/10.3390/polym15102256 . 10.3390/polym15102256 . Polymers
A standardised protocol for blood and cerebrospinal fluid collection and processing for biomarker research in ataxia
Santana, M.M.; Gaspar, L.S.; Pinto, M.M.; Silva, P.; Adão, D.; Pereira, D.; Ribeiro, J.A.; et al, 2023. Neuropathology and Applied Neurobiology. 2. 49. 2023. http://www.scopus.com/inward/record.url?eid=2-s2.0-85153765323&partnerID=MN8TOARS . 10.1111/nan.12892 . Neuropathology and Applied Neurobiology
SCAview: an Intuitive Visual Approach to the Integrative Analysis of Clinical Data in Spinocerebellar Ataxias
Uebachs, M.; Wegner, P.; Schaaf, S.; Kugai, S.; Jacobi, H.; Kuo, S.-H.; Ashizawa, T.; et al, 2023. Cerebellum. 2023. http://www.scopus.com/inward/record.url?eid=2-s2.0-85151467845&partnerID=MN8TOARS . 10.1007/s12311-023-01546-0 . Cerebellum
Extracellular vesicle-based delivery of silencing sequences for the treatment of Machado-Joseph disease/spinocerebellar ataxia type 3
Rufino-Ramos, D.; Albuquerque, P.R.; Leandro, K.; Carmona, V.; Martins, I.M.; Fernandes, R.; Henriques, C.; et al, 2023. Molecular Therapy. 1275 - 1292. 5. 31. 2023. http://www.scopus.com/inward/record.url?eid=2-s2.0-85154619372&partnerID=MN8TOARS . 10.1016/j.ymthe.2023.04.001 . Molecular Therapy
The stress granule protein G3BP1 alleviates spinocerebellar ataxia-associated deficits
Koppenol, R.; Conceição, A.; Afonso, I.T.; Afonso-Reis, R.; Costa, R.G.; Tomé, S.; Teixeira, D.; et al, 2023. Brain. 2346 - 2363. 6. 146. 2023. http://www.scopus.com/inward/record.url?eid=2-s2.0-85160404450&partnerID=MN8TOARS . 10.1093/brain/awac473 . Brain
Ghrelin delays premature aging in Hutchinson-Gilford progeria syndrome
Ferreira-Marques, M.; Carvalho, A.; Franco, A.C.; Leal, A.; Botelho, M.; Carmo-Silva, S.; Águas, R.; et al, 2023. Aging Cell. 2023. http://www.scopus.com/inward/record.url?eid=2-s2.0-85174420928&partnerID=MN8TOARS . 10.1111/acel.13983 . Aging Cell
Correction to: The frequency of non-motor symptoms in SCA3 and their association with disease severity and lifestyle factors
Hengel, Holger; Martus, Peter; Faber, Jennifer; Giunti, Paola; Garcia-Moreno, Hector; Solanky, Nita; Klockgether, Thomas; et al, 2023. Journal of Neurology. 628 - 629. 1. 271. 2023. http://dx.doi.org/10.1007/s00415-023-12064-8 . 10.1007/s00415-023-12064-8 . Journal of Neurology
Identification of the calpain-generated toxic fragment of ataxin-3 protein provides new avenues for therapy of Machado–Joseph disease| Spinocerebellar ataxia type 3
Ana Teresa Simões; Vítor Carmona; Joana Duarte-Neves; Janete Cunha-Santos; Luís Pereira de Almeida, 2022. Neuropathology and Applied Neurobiology. 2022. https://doi.org/10.1111/nan.12748 . 10.1111/nan.12748 . Neuropathology and Applied Neurobiology
Establishment and characterization of human pluripotent stem cells-derived brain organoids to model cerebellar diseases
Brás, J.; Henriques, D.; Moreira, R.; Santana, M.M.; Silva-Pedrosa, R.; Adão, D.; Braz, S.; et al, 2022. Scientific Reports. 1. 12. 2022. http://www.scopus.com/inward/record.url?eid=2-s2.0-85134499812&partnerID=MN8TOARS . 10.1038/s41598-022-16369-y . Scientific Reports
Deciphering a new role for Ataxin-2 in the hypothalamus: linking metabolism and circadian rhythm regulation.
Carmo-Silva, Sara; Ferreira-Marques, Marisa; Nóbrega, Clévio David Rodrigues ; Botelho, Mariana; Costa, Daniela G.; Pulst, Stefan M; Pereira de Almeida, Luis Fernando Morgado; Cavadas, Claudia, 2022. Molecular Endocrinology. 2022. under revision Molecular Endocrinology
ULK overexpression mitigates motor deficits and neuropathology in mouse models of Machado-Joseph disease
Vasconcelos-Ferreira, A.; Martins, I.M.; Lobo, D.; Pereira, D.; Lopes, M.M.; Faro, R.; Lopes, S.M.; et al, 2022. Molecular Therapy. 370 - 387. 1. 30. 2022. http://www.scopus.com/inward/record.url?eid=2-s2.0-85115030983&partnerID=MN8TOARS . 10.1016/j.ymthe.2021.07.012 . Molecular Therapy
Using genetically modified extracellular vesicles as a non-invasive strategy to evaluate brain-specific cargo
Rufino-Ramos, D.; Lule, S.; Mahjoum, S.; Ughetto, S.; Cristopher Bragg, D.; Pereira de Almeida, L.; Breakefield, X.O.; Breyne, K., 2022. Biomaterials. 281. 2022. http://www.scopus.com/inward/record.url?eid=2-s2.0-85122652441&partnerID=MN8TOARS . 10.1016/j.biomaterials.2022.121366 . Biomaterials
MiRNA-Mediated Knockdown of ATXN3 Alleviates Molecular Disease Hallmarks in a Mouse Model for Spinocerebellar Ataxia Type 3
Nobre, R.J.; Lobo, D.D.; Henriques, C.; Duarte, S.P.; Lopes, S.M.; Silva, A.C.; Lopes, M.M.; et al, 2022. Nucleic Acid Therapeutics. 194 - 205. 3. 32. 2022. http://www.scopus.com/inward/record.url?eid=2-s2.0-85129129262&partnerID=MN8TOARS . 10.1089/nat.2021.0020 . Nucleic Acid Therapeutics
Cerebellar morphometric and spectroscopic biomarkers for Machado-Joseph Disease
Miranda, C.O.; Nobre, R.J.; Paiva, V.H.; Duarte, J.V.; Castelhano, J.; Petrella, L.I.; Sereno, J.; et al, 2022. Acta Neuropathologica Communications. 1. 10. 2022. http://www.scopus.com/inward/record.url?eid=2-s2.0-85126741777&partnerID=MN8TOARS . 10.1186/s40478-022-01329-4 . Acta Neuropathologica Communications
Characterization of Lifestyle in Spinocerebellar Ataxia Type 3 and Association with Disease Severity
Holger Hengel; Peter Martus; Jennifer Faber; Hector Garcia-Moreno; Nita Solanky; Paola Giunti; Thomas Klockgether; et al, 2022. Movement Disorders. 2022. https://doi.org/10.1002/mds.28844 . 10.1002/mds.28844 . Movement Disorders
Photodynamic disinfection of SARS-CoV-2 clinical samples using a methylene blue formulation
Lobo, C.S.; Rodrigues-Santos, P.; Pereira, D.; Núñez, J.; Trêpa, J.C.D.; Sousa, D.L.; Lourenço, J.V.; et al, 2022. Photochemical and Photobiological Sciences. 2022. http://www.scopus.com/inward/record.url?eid=2-s2.0-85126514875&partnerID=MN8TOARS . 10.1007/s43630-022-00202-6 . Photochemical and Photobiological Sciences
A new protocol for whole-brain biodistribution analysis of AAVs by tissue clearing, light-sheet microscopy and semi-automated spatial quantification
Miguel M. Lopes; Jacques Paysan; José Rino; Sara M. Lopes; Luís Pereira de Almeida; Luísa Cortes; Rui Jorge Nobre, 2022. Gene Therapy. 2022. https://doi.org/10.1038/s41434-022-00372-z . 10.1038/s41434-022-00372-z . Gene Therapy
Differential Temporal Dynamics of Axial and Appendicular Ataxia in SCA3
Roderick P.P.W.M. Maas; Steven Teerenstra; Manuela Lima; Paula Pires; Luís Pereira de Almeida; Judith van Gaalen; Dagmar Timmann; et al, 2022. Movement Disorders. 2022. https://doi.org/10.1002/mds.29135 . 10.1002/mds.29135 . Movement Disorders
SIRT2 Deficiency Exacerbates Hepatic Steatosis via a Putative Role of the ER Stress Pathway
Helena Leal; João Cardoso; Patrícia Valério; Marta Quatorze; Vítor Carmona; Janete Cunha-Santos; Luís Pereira de Almeida; et al, 2022. International Journal of Molecular Sciences. 6790 - 6790. 12. 23. 2022. https://doi.org/10.3390/ijms23126790 . 10.3390/ijms23126790 . International Journal of Molecular Sciences
Tau and neurofilament light-chain as fluid biomarkers in spinocerebellar ataxia type 3
Hector Garcia-Moreno; Mercedes Prudencio; Gilbert Thomas-Black; Nita Solanky; Karen R. Jansen-West; Rana Hanna AL-Shaikh; Amanda Heslegrave; et al, 2022. European Journal of Neurology. 2022. https://doi.org/10.1111/ene.15373 . 10.1111/ene.15373 . European Journal of Neurology
Exogenous loading of extracellular vesicles, virus-like particles, and lentiviral vectors with supercharged proteins
Koen Breyne; Stefano Ughetto; David Rufino-Ramos; Shadi Mahjoum; Emily A. Grandell; Luís P. de Almeida; Xandra O. Breakefield, 2022. Communications Biology. 2022. https://doi.org/10.1038/s42003-022-03440-7 . 10.1038/s42003-022-03440-7 . Communications Biology
Osteosarcoma-Derived Exosomes as Potential PET Imaging Nanocarriers for Lung Metastasis
Almeida, S.F.F.; Fonseca, A.; Sereno, J.; Ferreira, H.R.S.; Lapo-Pais, M.; Martins-Marques, T.; Rodrigues, T.; et al, 2022. Small. 49. 18. 2022. http://www.scopus.com/inward/record.url?eid=2-s2.0-85141363010&partnerID=MN8TOARS . 10.1002/smll.202203999 . Small
SIRT1 activation and its circadian clock control: a promising approach against (frailty in) neurodegenerative disorders
Rodrigo F. N. Ribeiro; Dina Pereira; Luis Pereira de Almeida; Maria Manuel C. Silva; Cláudia Cavadas, 2022. Aging Clinical and Experimental Research. 2022. https://doi.org/10.1007/s40520-022-02257-y . 10.1007/s40520-022-02257-y . Aging Clinical and Experimental Research
The autophagy-enhancing drug carbamazepine improves neuropathology and motor impairment in mouse models of Machado–Joseph disease
Ana Vasconcelos-Ferreira; Sara Carmo-Silva; José Miguel Codêsso; Patrick Silva; Alberto Rolim Muro Martinez; Marcondes Cavalcante França Jr; Clévio Nóbrega; et al, 2022. Neuropathology and Applied Neurobiology. 2022. https://doi.org/10.1111/nan.12763 . 10.1111/nan.12763 . Neuropathology and Applied Neurobiology
Editorial: Gene therapy 2.0: Biotechnology for circuit engineering and complex therapeutic approaches
Quintino, L.; Almeida, L.P.D.; Lundberg, C., 2022. Frontiers in Bioengineering and Biotechnology. 10. 2022. http://www.scopus.com/inward/record.url?eid=2-s2.0-85143363515&partnerID=MN8TOARS . 10.3389/fbioe.2022.1082883 . Frontiers in Bioengineering and Biotechnology
Highly Porous Composite Scaffolds Endowed with Antibacterial Activity for Multifunctional Grafts in Bone Repair
Ana S. Neto; Patrícia Pereira; Ana C. Fonseca; Carla Dias; Mariana C. Almeida; Inês Barros; Catarina O. Miranda; et al, 2021. Polymers. 2021. https://doi.org/10.3390/polym13244378 . 10.3390/polym13244378 . Polymers
Neuropeptide Y (NPY) intranasal delivery alleviates Machado–Joseph disease
Duarte-Neves, J.; Cavadas, C.; Pereira de Almeida, L., 2021. Scientific Reports. 1. 11. 2021. http://www.scopus.com/inward/record.url?eid=2-s2.0-85100703911&partnerID=MN8TOARS . 10.1038/s41598-021-82339-5 . Scientific Reports
Mesenchymal stromal cells to fight SARS-CoV-2: Taking advantage of a pleiotropic therapy
Barros, I.; Silva, A.; de Almeida, L.P.; Miranda, C.O., 2021. Cytokine and Growth Factor Reviews. 2021. http://www.scopus.com/inward/record.url?eid=2-s2.0-85098642605&partnerID=MN8TOARS . 10.1016/j.cytogfr.2020.12.002 . Cytokine and Growth Factor Reviews
Guidelines for the use and interpretation of assays for monitoring autophagy (4th edition)
Klionsky, D.J.; Abdel-Aziz, A.K.; Abdelfatah, S.; Abdellatif, M.; Abdoli, A.; Abel, S.; Abeliovich, H.; et al, 2021. Autophagy. 2021. http://www.scopus.com/inward/record.url?eid=2-s2.0-85102619204&partnerID=MN8TOARS . 10.1080/15548627.2020.1797280 . Autophagy
Stress granules, RNA-binding proteins and polyglutamine diseases: too much aggregation?
Marcelo, A.; Koppenol, R.; de Almeida, L.P.; Matos, C.A.; Nóbrega, C., 2021. Cell Death and Disease. 6. 12. 2021. http://www.scopus.com/inward/record.url?eid=2-s2.0-85107594482&partnerID=MN8TOARS . 10.1038/s41419-021-03873-8 . Cell Death and Disease
Living Proof of Activity of Extracellular Vesicles in the Central Nervous System
Shadi Mahjoum; David Rufino-Ramos; Luís Pereira de Almeida; Marike L. D. Broekman; Xandra O. Breakefield; Thomas S. van Solinge; Mahjoum, Shadi; et al, 2021. International Journal of Molecular Sciences. 7294 - 7294. 14. 22. 2021. https://doi.org/10.3390/ijms22147294 . 10.3390/ijms22147294 . International Journal of Molecular Sciences
Extracellular vesicles physiological role and the particular case of disease-spreading mechanisms in polyglutamine diseases
Moreira, R.; Mendonça, L.S.; de Almeida, L.P., 2021. International Journal of Molecular Sciences. 22. 22. 2021. http://www.scopus.com/inward/record.url?eid=2-s2.0-85118955937&partnerID=MN8TOARS . 10.3390/ijms222212288 . International Journal of Molecular Sciences
Polyglutamine-Expanded Ataxin-3: A Target Engagement Marker for Spinocerebellar Ataxia Type 3 in Peripheral Blood
Hübener-Schmid, J.; Kuhlbrodt, K.; Peladan, J.; Faber, J.; Santana, M.M.; Hengel, H.; Jacobi, H.; et al, 2021. Movement Disorders. 2021. http://www.scopus.com/inward/record.url?eid=2-s2.0-85113149446&partnerID=MN8TOARS . 10.1002/mds.28749 . Movement Disorders
Autophagy in Spinocerebellar ataxia type 2, a dysregulated pathway, and a target for therapy
Marcelo, A.; Afonso, I.T.; Afonso-Reis, R.; Brito, D.V.C.; Costa, R.G.; Rosa, A.; Alves-Cruzeiro, J.; et al, 2021. Cell Death and Disease. 8 - 28. 12. 12. 2021. http://www.scopus.com/inward/record.url?eid=2-s2.0-85120082938&partnerID=MN8TOARS . 10.1038/s41419-021-04404-1 . Cell Death and Disease
The Parkinson's-disease-associated mutation LRRK2-G2019S alters dopaminergic differentiation dynamics via NR2F1
Walter, J.; Bolognin, S.; Poovathingal, S.K.; Magni, S.; Gérard, D.; Antony, P.M.A.; Nickels, S.L.; et al, 2021. Cell Reports. 3. 37. 2021. http://www.scopus.com/inward/record.url?eid=2-s2.0-85117275453&partnerID=MN8TOARS . 10.1016/j.celrep.2021.109864 . Cell Reports
Highly specific blood-brain barrier transmigrating single-domain antibodies selected by an in vivo phage display screening
Aguiar, S.I.; Dias, J.N.R.; André, A.S.; Silva, M.L.; Martins, D.; Carrapiço, B.; Castanho, M.; et al, 2021. Pharmaceutics. 10. 13. 2021. http://www.scopus.com/inward/record.url?eid=2-s2.0-85116780736&partnerID=MN8TOARS . 10.3390/pharmaceutics13101598 . Pharmaceutics
Neuropeptide Y Enhances Progerin Clearance and Ameliorates the Senescent Phenotype of Human Hutchinson-Gilford Progeria Syndrome Cells
Aveleira, Célia A; Ferreira-Marques, Marisa; Cortes, Luísa; Valero, Jorge; Pereira, Dina; Pereira de Almeida, Luís; Cavadas, Cláudia, 2020. The Journals of Gerontology: Series A. 2020. http://dx.doi.org/10.1093/gerona/glz280 . 10.1093/gerona/glz280 . The Journals of Gerontology: Series A
Simple and Fast Size Exclusion Chromatography-based Protocol to Isolate Human Plasma-derived Extracellular Vesicles for Transcriptional Research
Gaspar, Laetitia S; Magda M Santana; Carina Henriques; Pinto, Maria Manuel ; Teresa M Ribeiro Rodrigues; Girao, Henrique; Rui Jorge Nobre; Pereira de Almeida, Luis , 2020. Molecular Therapy - Methods & Clinical Development . 2020. http://dx.doi.org/10.1016/j.omtm.2020.07.012. 10.1016/j.omtm.2020.07.012 . published Molecular Therapy - Methods & Clinical Development
The blood-brain barrier is disrupted in Machado-Joseph disease/spinocerebellar ataxia type 3: evidence from transgenic mice and human post-mortem samples
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Simple and Fast SEC-Based Protocol to Isolate Human Plasma-Derived Extracellular Vesicles for Transcriptional Research
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Protocol for the Characterization of the CAG Tract and Flanking Polymorphisms in Machado-Joseph Disease: Impact on Diagnosis and Development of Gene-Based Therapies
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miRNA-31 Improves Cognition and Abolishes Amyloid-ß Pathology by Targeting APP and BACE1 in an Animal Model of Alzheimer’s Disease
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Mitochondrial and Redox Modifications in Huntington Disease Induced Pluripotent Stem Cells Rescued by CRISPR/Cas9 CAGs Targeting
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Mesenchymal Stromal Cells’ Therapy for Polyglutamine Disorders: Where Do We Stand and Where Should We Go?
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Neurofilaments in spinocerebellar ataxia type 3: blood biomarkers at the preataxic and ataxic stage in humans and mice
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Reversal of mitochondrial abnormalities by CRISPR/Cas9 editing of the mutant HTT allele in vitro in Huntington´s Disease patient-derived pluripotent and neural stem cells
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Restoring brain cholesterol turnover improves autophagy and has therapeutic potential in mouse models of spinocerebellar ataxia
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Neural Stem Cells of Parkinson's Disease Patients Exhibit Aberrant Mitochondrial Morphology and Functionality.
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RNA interference therapy for Machado-Joseph disease: Long-term safety profile of lentiviral vectors encoding short hairpin RNAs targeting mutant ataxin-3
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Antisense oligonucleotide therapeutics in neurodegenerative diseases: the case of polyglutamine disorders
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Successes and Hurdles in Stem Cells Application and Production for Brain Transplantation
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Cordycepin activates autophagy through AMPK phosphorylation to reduce abnormalities in Machado-Joseph disease models
Marcelo, Adriana; Brito, Filipa; Carmo-Silva, Sara; Matos, Carlos A.; Alves-Cruzeiro, Joao; Vasconcelos-Ferreira, Ana; Koppenol, Rebekah; et al, 2019. Human molecular genetics. 51 - 63. 1. 28. 2019. http://hdl.handle.net/10400.1/14482 . 10.1093/hmg/ddy328 . Human molecular genetics
Ibuprofen enhances synaptic function and neural progenitors proliferation markers and improves neuropathology and motor coordination in Machado-Joseph disease models
Liliana S Mendonça; Clévio Nóbrega; Silvia Tavino; Maximilian Brinkhaus; Carlos Matos; Sandra Tomé; Ricardo Moreira; et al, 2019. Human Molecular Genetics. 2019. https://doi.org/10.1093/hmg/ddz097 . 10.1093/hmg/ddz097 . Human Molecular Genetics
Repeated Mesenchymal Stromal Cell Treatment Sustainably Alleviates Machado-Joseph Disease
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Loss of hierarchical imprinting regulation at the Prader-Willi/Angelman syndrome locus in human iPSCs
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Dipeptidyl peptidase IV (DPP-IV) inhibition prevents fibrosis in adipose tissue of obese mice
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Stroma-derived IL-6, G-CSF and Activin-A mediated dedifferentiation of lung carcinoma cells into cancer stem cells
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Control of Huntington's Disease-Associated Phenotypes by the Striatum-Enriched Transcription Factor Foxp2
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Generation of human iPSC line from a patient with laterality defects and associated congenital heart anomalies carrying a DAND5 missense alteration
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Extracellular vesicles: Novel promising delivery systems for therapy of brain diseases
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Caffeine alleviates progressive motor deficits in a transgenic mouse model of spinocerebellar ataxia
Gonçalves, N.; Simões, A.T.; Prediger, R.D.; Hirai, H.; Cunha, R.A.; Pereira de Almeida, L., 2017. Annals of Neurology. 407 - 418. 3. 81. 2017. http://www.scopus.com/inward/record.url?eid=2-s2.0-85013441188&partnerID=MN8TOARS . 10.1002/ana.24867 . Annals of Neurology
Proteolytic cleavage of polyglutamine disease-causing proteins: Revisiting the toxic fragment hypothesis
Matos, C.A.; de Almeida, L.P.; Nóbrega, C., 2017. Current Pharmaceutical Design. 753 - 775. 5. 23. 2017. http://www.scopus.com/inward/record.url?eid=2-s2.0-85017122161&partnerID=MN8TOARS . 10.2174/1381612822666161227121912 . Current Pharmaceutical Design
Unravelling Endogenous MicroRNA System Dysfunction as a New Pathophysiological Mechanism in Machado-Joseph Disease
Carmona, V.; Cunha-Santos, J.; Onofre, I.; Simões, A.T.; Vijayakumar, U.; Davidson, B.L.; Pereira de Almeida, L., 2017. Molecular Therapy. 1038 - 1055. 4. 25. 2017. http://www.scopus.com/inward/record.url?eid=2-s2.0-85016966764&partnerID=MN8TOARS . 10.1016/j.ymthe.2017.01.021 . Molecular Therapy
Unraveling the Role of Ataxin-2 in Metabolism
Carmo-Silva, S.; Nobrega, C.; Pereira de Almeida, L.; Cavadas, C., 2017. Trends in Endocrinology and Metabolism. 309 - 318. 4. 28. 2017. http://www.scopus.com/inward/record.url?eid=2-s2.0-85009726744&partnerID=MN8TOARS . 10.1016/j.tem.2016.12.006 . Trends in Endocrinology and Metabolism
Gene therapy for the CNS using AAVs: The impact of systemic delivery by AAV9
Saraiva, J.; Nobre, R.J.; Pereira de Almeida, L., 2016. Journal of Controlled Release. 94 - 109. 241. 2016. http://www.scopus.com/inward/record.url?eid=2-s2.0-84988451695&partnerID=MN8TOARS . 10.1016/j.jconrel.2016.09.011 . Journal of Controlled Release
Adenosine A<inf>2A</inf> receptors in the amygdala control synaptic plasticity and contextual fear memory
Simões, A.P.; MacHado, N.J.; Gonçalves, N.; Kaster, M.P.; Simões, A.T.; Nunes, A.; Pereira De Almeida, L.; et al, 2016. Neuropsychopharmacology. 2862 - 2871. 12. 41. 2016. http://www.scopus.com/inward/record.url?eid=2-s2.0-84978777019&partnerID=MN8TOARS . 10.1038/npp.2016.98 . Neuropsychopharmacology
Intravenous administration of brain-targeted stable nucleic acid lipid particles alleviates Machado-Joseph disease neurological phenotype
Conceição, M.; Mendonça, L.; Nóbrega, C.; Gomes, C.; Costa, P.; Hirai, H.; Moreira, J.N.; et al, 2016. Biomaterials. 124 - 137. 82. 2016. http://www.scopus.com/inward/record.url?eid=2-s2.0-84956644943&partnerID=MN8TOARS . 10.1016/j.biomaterials.2015.12.021 . Biomaterials
Safety profile of the intravenous administration of brain-targeted stable nucleic acid lipid particles
Conceição, M.; Mendonça, L.; Nóbrega, C.; Gomes, C.; Costa, P.; Hirai, H.; Moreira, J.N.; et al, 2016. Data in Brief. 700 - 705. 6. 2016. http://www.scopus.com/inward/record.url?eid=2-s2.0-84956617357&partnerID=MN8TOARS . 10.1016/j.dib.2016.01.017 . Data in Brief
Gephyrin Cleavage in In Vitro Brain Ischemia Decreases GABA<inf>A</inf> Receptor Clustering and Contributes to Neuronal Death
Costa, J.T.; Mele, M.; Baptista, M.S.; Gomes, J.R.; Ruscher, K.; Nobre, R.J.; de Almeida, L.P.; Wieloch, T.; Duarte, C.B., 2016. Molecular Neurobiology. 3513 - 3527. 6. 53. 2016. http://www.scopus.com/inward/record.url?eid=2-s2.0-84931863889&partnerID=MN8TOARS . 10.1007/s12035-015-9283-2 . Molecular Neurobiology
Motor dysfunctions and neuropathology in mouse models of spinocerebellar ataxia type 2: A comprehensive review
Da Conceição Alves-Cruzeiro, J.M.; Mendonça, L.; De Almeida, L.P.; Nóbrega, C., 2016. Frontiers in Neuroscience. DEC. 10. 2016. http://www.scopus.com/inward/record.url?eid=2-s2.0-85009729313&partnerID=MN8TOARS . 10.3389/fnins.2016.00572 . Frontiers in Neuroscience
Ataxin-3 phosphorylation decreases neuronal defects in spinocerebellar ataxia type 3 models
Matos, C.A.; Nóbrega, C.; Louros, S.R.; Almeida, B.; Ferreiro, E.; Valero, J.; De Almeida, L.P.; Macedo-Ribeiro, S.; Carvalho, A.L., 2016. Journal of Cell Biology. 465 - 480. 4. 212. 2016. http://www.scopus.com/inward/record.url?eid=2-s2.0-84959536774&partnerID=MN8TOARS . 10.1083/jcb.201506025 . Journal of Cell Biology
Caloric restriction stimulates autophagy in rat cortical neurons through neuropeptide Y and ghrelin receptors activation
Ferreira-Marques, M.; Aveleira, C.A.; Carmo-Silva, S.; Botelho, M.; de Almeida, L.P.; Cavadas, C., 2016. Aging. 1470 - 1484. 7. 8. 2016. http://www.scopus.com/inward/record.url?eid=2-s2.0-85014143445&partnerID=MN8TOARS . 10.18632/aging.100996 . Aging
Neuropeptide Y (NPY) as a therapeutic target for neurodegenerative diseases
Duarte-Neves, J.; Pereira de Almeida, L.; Cavadas, C., 2016. Neurobiology of Disease. 210 - 214. 95. 2016. http://www.scopus.com/inward/record.url?eid=2-s2.0-84979934564&partnerID=MN8TOARS . 10.1016/j.nbd.2016.07.022 . Neurobiology of Disease
Fibroblasts of Machado Joseph Disease patients reveal autophagy impairment
Onofre, I.; Mendonça, N.; Lopes, S.; Nobre, R.; De Melo, J.B.; Carreira, I.M.; Januário, C.; Gonçalves, A.F.; De Almeida, L.P., 2016. Scientific Reports. 6. 2016. http://www.scopus.com/inward/record.url?eid=2-s2.0-84975709020&partnerID=MN8TOARS . 10.1038/srep28220 . Scientific Reports
Caloric restriction blocks neuropathology and motor deficits in Machado-Joseph disease mouse models through SIRT1 pathway
Cunha-Santos, J.; Duarte-Neves, J.; Carmona, V.; Guarente, L.; De Almeida, L.P.; Cavadas, C., 2016. Nature Communications. 7. 2016. http://www.scopus.com/inward/record.url?eid=2-s2.0-84968928647&partnerID=MN8TOARS . 10.1038/ncomms11445 . Nature Communications
Neuropeptide Y mitigates neuropathology and motor deficits in mouse models of Machado-Joseph disease
Duarte-Neves, J.; Gonçalves, N.; Cunha-Santos, J.; Simões, A.T.; den Dunnen, W.F.A.; Hirai, H.; Kügler, S.; Cavadas, C.; de Almeida, L.P., 2015. Human Molecular Genetics. 5451 - 5463. 19. 24. 2015. http://www.scopus.com/inward/record.url?eid=2-s2.0-84943793236&partnerID=MN8TOARS . 10.1093/hmg/ddv271 . Human Molecular Genetics
Neuropeptide Y stimulates autophagy in hypothalamic neurons
Aveleira, C.A.; Botelho, M.; Carmo-Silva, S.; Pascoal, J.F.; Ferreira-Marques, M.; Nóbrega, C.; Cortes, L.; et al, 2015. Proceedings of the National Academy of Sciences of the United States of America. E1642 - E1651. 13. 112. 2015. http://www.scopus.com/inward/record.url?eid=2-s2.0-84926180461&partnerID=MN8TOARS . 10.1073/pnas.1416609112 . Proceedings of the National Academy of Sciences of the United States of America
MiRNA-21 silencing mediated by tumor-targeted nanoparticles combined with sunitinib: A new multimodal gene therapy approach for glioblastoma
Costa, P.M.; Cardoso, A.L.; Custódia, C.; Cunha, P.; Pereira De Almeida, L.; Pedroso De Lima, M.C., 2015. Journal of Controlled Release. 31 - 39. 207. 2015. http://www.scopus.com/inward/record.url?eid=2-s2.0-84926665383&partnerID=MN8TOARS . 10.1016/j.jconrel.2015.04.002 . Journal of Controlled Release
Re-establishing ataxin-2 downregulates translation of mutant ataxin-3 and alleviates Machado-Joseph disease
Nóbrega, C.; Carmo-Silva, S.; Albuquerque, D.; Vasconcelos-Ferreira, A.; Vijayakumar, U.-G.; Mendonça, L.; Hirai, H.; Pereira De Almeida, L., 2015. Brain. 3537 - 3554. 12. 138. 2015. http://www.scopus.com/inward/record.url?eid=2-s2.0-84951031850&partnerID=MN8TOARS . 10.1093/brain/awv298 . Brain
Transplantation of cerebellar neural stem cells improves motor coordination and neuropathology in Machado-Joseph disease mice
Mendonça, L.S.; Nóbrega, C.; Hirai, H.; Kaspar, B.K.; De Almeida, L.P., 2015. Brain. 320 - 335. 2. 138. 2015. http://www.scopus.com/inward/record.url?eid=2-s2.0-84922366384&partnerID=MN8TOARS . 10.1093/brain/awu352 . Brain
Early miR-155 upregulation contributes to neuroinflammation in Alzheimer's disease triple transgenic mouse model
Guedes, J.R.; Custódia, C.M.; Silva, R.J.; de Almeida, L.P.; de Lima, M.C.P.; Cardoso, A.L., 2014. Human Molecular Genetics. 6286 - 6301. 23. 23. 2014. http://www.scopus.com/inward/record.url?eid=2-s2.0-84926341070&partnerID=MN8TOARS . 10.1093/hmg/ddu348 . Human Molecular Genetics
Calpain inhibition reduces ataxin-3 cleavage alleviating neuropathology and motor impairments in mouse models of machado-Joseph disease
Simões, A.T.; Gonçalves, N.; Nobre, R.J.; Duarte, C.B.; Pereira de Almeida, L., 2014. Human Molecular Genetics. 4932 - 4944. 18. 23. 2014. http://www.scopus.com/inward/record.url?eid=2-s2.0-84906821927&partnerID=MN8TOARS . 10.1093/hmg/ddu209 . Human Molecular Genetics
Let-7 coordinately suppresses components of the amino acid sensing pathway to repress mTORC1 and induce autophagy
Dubinsky, A.N.; Dastidar, S.G.; Hsu, C.L.; Zahra, R.; Djakovic, S.N.; Duarte, S.; Esau, C.C.; et al, 2014. Cell Metabolism. 626 - 638. 4. 20. 2014. http://www.scopus.com/inward/record.url?eid=2-s2.0-84907967415&partnerID=MN8TOARS . 10.1016/j.cmet.2014.09.001 . Cell Metabolism
Fluoxetine induces proliferation and inhibits differentiation of hypothalamic neuroprogenitor cells in vitro
Sousa-Ferreira, L.; Aveleira, C.; Botelho, M.; Álvaro, A.R.; Pereira De Almeida, L.; Cavadas, C., 2014. PLoS ONE. 3. 9. 2014. http://www.scopus.com/inward/record.url?eid=2-s2.0-84897138484&partnerID=MN8TOARS . 10.1371/journal.pone.0088917 . PLoS ONE
RNA interference mitigates motor and neuropathological deficits in a cerebellar mouse model of Machado-Joseph disease
Nóbrega, C.; Nascimento-Ferreira, I.; Onofre, I.; Albuquerque, D.; Déglon, N.; De Almeida, L.P., 2014. PLoS ONE. 8. 9. 2014. http://www.scopus.com/inward/record.url?eid=2-s2.0-84929055238&partnerID=MN8TOARS . 10.1371/journal.pone.0100086 . PLoS ONE
Silencing Mutant Ataxin-3 Rescues Motor Deficits and Neuropathology in Machado-Joseph Disease Transgenic Mice
Nóbrega, Clévio; Nascimento-Ferreira, Isabel; Onofre, Isabel; Albuquerque, David; Hirai, Hirokazu; Déglon, Nicole; de Almeida, Luís Pereira; Chiesa, Roberto, 2013. PLoS ONE. e52396 - e52396. 1. 8. 2013. http://www.plosone.org/article/info%3Adoi%2F10.1371%2Fjournal.pone.0052396 . 10.1371/journal.pone.0052396 . published PLoS ONE
IGF-1 Intranasal Administration Rescues Huntington's Disease Phenotypes in YAC128 Mice
Lopes, Carla; Ribeiro, Márcio; Duarte, Ana I.; Humbert, Sandrine; Saudou, Frederic; Pereira de Almeida, Luís; Hayden, Michael; Rego, A. Cristina, 2013. Molecular Neurobiology. 0 - 0. 0. 2013. 10.1007/s12035-013-8585-5 . published Molecular Neurobiology
Role of hypothalamic neurogenesis in feeding regulation
Sousa-Ferreira, Lígia; Almeida, Luís Pereira de; Cavadas, Cláudia, 2013. Trends in Endocrinology & Metabolism. 1 - 9. .. 2013. http://www.cell.com/trends/endocrinology-metabolism//retrieve/pii/S1043276013001744?_returnURL=http://linkinghub.elsevier.com/retrieve/pii/S1043276013001744?showall=true . 10.1016/j.tem.2013.10.005 . published Trends in Endocrinology & Metabolism
Beclin 1 mitigates motor and neuropathological deficits in genetic mouse models of Machado-Joseph disease
Nascimento-Ferreira, I.; Nobrega, C.; Vasconcelos-Ferreira, A.; Onofre, I.; Albuquerque, D.; Aveleira, C.; Hirai, H.; Deglon, N.; Pereira de Almeida, L, 2013. Brain. 2173 - 2188. 7. 136. 2013. http://brain.oxfordjournals.org/cgi/content/full/awt144? ijkey=5Fgyaqt9oIUEAui&keytype=ref . 10.1093/brain/awt144 . published Brain
Caffeine and adenosine A2A receptor inactivation decrease striatal neuropathology in a lentiviral-based model of Machado-Joseph disease
Gonçalves, Nélio; Simões, Ana T.; Cunha, Rodrigo A.; de Almeida, Luís Pereira; Almeida, Luís Pereira de, 2013. Annals of Neurology. 655 - 666. 5. 73. 2013. http://hdl.handle.net/10316/28116 . 10.1002/ana.23866 . published Annals of Neurology
miR-155 modulates microglia-mediated immune response by down-regulating SOCS-1 and promoting cytokine and nitric oxide production
Cardoso, Ana L.; Guedes, Joana R.; Pereira de Almeida, Luís; Pedroso de Lima, Maria C., 2012. Immunology. 73 - 88. 1. 135. 2012. 10.1111/j.1365-2567.2011.03514.x . published Immunology
MicroRNA-21 silencing enhances the cytotoxic effect of the antiangiogenic drug sunitinib in glioblastoma
Costa, P. M.; Cardoso, A. L.; Nobrega, C.; Pereira de Almeida, L. F.; Bruce, J. N.; Canoll, P.; Pedroso de Lima, M. C., 2012. Human Molecular Genetics. 0 - 0. 0. 2012. 10.1093/hmg/dds496 . published Human Molecular Genetics
Overexpression of Mutant Ataxin-3 in Mouse Cerebellum Induces Ataxia and Cerebellar Neuropathology
Nóbrega, Clévio; Nascimento-Ferreira, Isabel; Onofre, Isabel; Albuquerque, David; Conceição, Mariana; Déglon, Nicole; Almeida, Luís Pereira, 2012. The Cerebellum. 0 - 0. 0. 2012. 10.1007/s12311-012-0432-0 . published The Cerebellum
PDGF-B-mediated downregulation of miR-21: new insights into PDGF signaling in glioblastoma
Costa, P. M.; Cardoso, A. L.; Pereira de Almeida, L. F.; Bruce, J. N.; Canoll, P.; Pedroso de Lima, M. C., 2012. Human Molecular Genetics. 0 - 0. 0. 0. 2012. 10.1093/hmg/dds358 . published Human Molecular Genetics
Células Estaminais Pluripotentes Induzidas (IPSCS) – A mais recente revolução das ciências da vida e da saúde
Pereira de Almeida, Luis Fernando Morgado, 2011. BOLETIM DO CIM - ROF 97 Mar/Abr 2011. 5 - 6. 97. 2011. published BOLETIM DO CIM - ROF 97 Mar/Abr 2011
Proliferative Hypothalamic Neurospheres Express NPY, AGRP, POMC, CART and Orexin-A and Differentiate to Functional Neurons
Sousa-Ferreira, Lígia; ¿?lvaro, Ana Rita; Aveleira, Célia; Santana, Magda; Brandão, Inês; Kügler, Sebastian; Pereira de Almeida, Luís; Cavadas, Cláudia; Linden, Rafael, 2011. PLoS ONE. 19745 - 19745. 5. 6. 2011. 10.1371/journal.pone.0019745 . published PLoS ONE
Moderate Long-Term Modulation of Neuropeptide Y in Hypothalamic Arcuate Nucleus Induces Energy Balance Alterations in Adult Rats
Sousa-Ferreira, Lígia; Garrido, Manuel; Nascimento-Ferreira, Isabel; Nobrega, Clévio; Santos-Carvalho, Ana; ¿?lvaro, Ana Rita; Rosmaninho-Salgado, Joana; et al, 2011. PLoS ONE. 22333 - 22333. 7. 6. 2011. 10.1371/journal.pone.0022333 . published PLoS ONE
Cleavage of the Vesicular GABA Transporter under Excitotoxic Conditions Is Followed by Accumulation of the Truncated Transporter in Nonsynaptic Sites
Gomes, J. R.; Lobo, A. C.; Melo, C. V.; Inacio, A. R.; Takano, J.; Iwata, N.; Saido, T. C.; et al, 2011. Journal of Neuroscience. 4622 - 4635. 12. 31. 2011. 10.1523/JNEUROSCI.3541-10.2011 . published Journal of Neuroscience
Gene Therapy for Parkinson's and Alzheimer's Diseases: from the Bench to Clinical Trials
Jorge Nobre, Rui; Pereira de Almeida, Luis, 2011. Current Pharmaceutical Design. 3434 - 3445. 31. 17. 2011. 10.2174/138161211798072472 . published Current Pharmaceutical Design
Overexpression of the autophagic beclin-1 protein clears mutant ataxin-3 and alleviates Machado-Joseph disease
Nascimento-Ferreira, I.; Santos-Ferreira, T.; Sousa-Ferreira, L.; Auregan, G.; Onofre, I.; Alves, S.; Dufour, N.; et al, 2011. Brain. 1400 - 1415. 5. 134. 2011. 10.1093/brain/awr047 . published Brain
Beclin-1 and autophagy: potential for new therapeutics in neurodegeneration
Pereira de Almeida, Luis Fernando Morgado; Nascimento-Ferreira, I., 2011. Neurodegenerative Disease Management. 349 - 351. 5. 1. 2011. http://www.futuremedicine.com/doi/full/10.2217/nmt.11.56 . published Neurodegenerative Disease Management
Tf-lipoplex-mediated c-Jun silencing improves neuronal survival following excitotoxic damage in vivo
Cardoso, A.L.C.; Costa, P.; de Almeida, L.P.; Simões, S.; Plesnila, N.; Culmsee, C.; Wagner, E.; Pedroso de Lima, M.C., 2010. Journal of Controlled Release. 392 - 403. 3. 142. 2010. 10.1016/j.jconrel.2009.11.004 . published Journal of Controlled Release
Silencing ataxin-3 mitigates degeneration in a rat model of Machado-Joseph disease: no role for wild-type ataxin-3?
Alves, S.; Nascimento-Ferreira, I.; Dufour, N.; Hassig, R.; Auregan, G.; Nobrega, C.; Brouillet, E.; et al, 2010. Human Molecular Genetics. 2380 - 2394. 12. 19. 2010. 10.1093/hmg/ddq111 . published Human Molecular Genetics
Characterization of common and rare human papillomaviruses in Portuguese women by the polymerase chain reaction, restriction fragment length polymorphism and sequencing
Nobre, Rui Jorge; Cruz, Eugénia; Real, Odete; de Almeida, Luís Pereira; Martins, Teresa C., 2010. Journal of Medical Virology. 1024 - 1032. 6. 82. 2010. 10.1002/jmv.21756 . published Journal of Medical Virology
Surfactants as Microbicides and Contraceptive Agents: A Systematic In Vitro Study
Vieira, Otilia V.; Hartmann, Diego O.; Cardoso, Carla M. P.; Oberdoerfer, Daniel; Baptista, Marta; Santos, Manuel A. S.; Almeida, Luis; et al, 2008. PLoS ONE. 2913 - 2913. 8. 3. 2008. 10.1371/journal.pone.0002913 . published PLoS ONE
Striatal and nigral pathology in a lentiviral rat model of Machado-Joseph disease
Alves, S.; Regulier, E.; Nascimento-Ferreira, I.; Hassig, R.; Dufour, N.; Koeppen, A.; Carvalho, A. L.; et al, 2008. Human Molecular Genetics. 2071 - 2083. 14. 17. 2008. 10.1093/hmg/ddn106 . published Human Molecular Genetics
Tf-lipoplexes for neuronal siRNA delivery: A promising system to mediate gene silencing in the CNS
Cardoso, A.L.C.; Simões, S.; de Almeida, L.P.; Plesnila, N.; Pedroso de Lima, M.C.; Wagner, E.; Culmsee, C., 2008. Journal of Controlled Release. 113 - 123. 2. 132. 2008. 10.1016/j.jconrel.2008.08.014 . published Journal of Controlled Release
Allele-Specific RNA Silencing of Mutant Ataxin-3 Mediates Neuroprotection in a Rat Model of Machado-Joseph Disease
Alves, Sandro; Nascimento-Ferreira, Isabel; Auregan, Gwennaëlle; Hassig, Raymonde; Dufour, Noëlle; Brouillet, Emmanuel; Pedroso de Lima, Maria C.; et al, 2008. PLoS ONE. 3341 - 3341. 10. 3. 2008. 10.1371/journal.pone.0003341 . published PLoS ONE
siRNA delivery by a transferrin-associated lipid-based vector: a non-viral strategy to mediate gene silencing
Cardoso, A. L. C.; Simões, S.; de Almeida, L. P.; Pelisek, J.; Culmsee, C.; Wagner, E.; Pedroso de Lima, M. C., 2007. The Journal of Gene Medicine. 170 - 183. 3. 9. 2007. 10.1002/jgm.1006 . published The Journal of Gene Medicine
Liposomal and Viral Vectors for Gene Therapy of the Central Nervous System
P. de Lima, M. C.; Girao da Cruz, M. T.; C. Cardoso, A. L; Simoes, S.; de Almeida, L. P., 2005. Current Drug Targets - CNS & Neurological Disorders. 453 - 465. 4. 4. 2005. 10.2174/1568007054546144 . published Current Drug Targets - CNS & Neurological Disorders
Tf-lipoplex-mediated NGF gene transfer to the CNS: neuronal protection and recovery in an excitotoxic model of brain injury
Teresa Girão da Cruz, M; Cardoso, A L C; de Almeida, L P; Simões, S; Pedroso de Lima, M C, 2005. Gene Therapy. 1242 - 1252. 16. 12. 2005. 10.1038/sj.gt.3302516 . published Gene Therapy
Molecular Targets and Therapeutic Strategies in Huntington's Disease
Rego, A. C.; de Almeida, L. P., 2005. Current Drug Targets - CNS & Neurological Disorders. 361 - 381. 4. 4. 2005. 10.2174/1568007054546081 . published Current Drug Targets - CNS & Neurological Disorders
Long-term lentiviral-mediated expression of ciliary neurotrophic factor in the striatum of Huntington's disease transgenic mice
Zala, Diana; Bensadoun, Jean-Charles; Pereira de Almeida, Luis; Leavitt, Blair R.; Gutekunst, Claire-Anne; Aebischer, Patrick; Hayden, Michael R.; Déglon, Nicole, 2004. Experimental Neurology. 26 - 35. 1. 185. 2004. http://www.sciencedirect.com/science/article/pii/S0014488603004436 . 10.1016/j.expneurol.2003.09.002 . published Experimental Neurology
Comparative study of GDNF delivery systems for the CNS: polymer rods, encapsulated cells, and lentiviral vectors
Bensadoun, Jean-Charles; Pereira de Almeida, Luis; Fine, Eric G; Tseng, Jack L; Déglon, Nicole; Aebischer, Patrick, 2003. Journal of Controlled Release. 107 - 115. 1-3. 87. 2003. 10.1016/S0168-3659(02)00353-X . published Journal of Controlled Release
Dose-Dependent Neuroprotective Effect of Ciliary Neurotrophic Factor Delivered via Tetracycline-Regulated Lentiviral Vectors in the Quinolinic Acid Rat Model of Huntington's Disease
Régulier, E.; Pereira de Almeida, L.; Sommer, B.; Aebischer, P.; Déglon, N., 2002. Human Gene Therapy. 1981 - 1990. 16. 13. 2002. 10.1089/10430340260355383 . published Human Gene Therapy
Lentiviral-Mediated Delivery of Mutant Huntingtin in the Striatum of Rats Induces a Selective Neuropathology Modulated by Polyglutamine Repeat Size, Huntingtin Expression Levels, and Protein Length
De Almeida, L.P.; Ross, C.A.; Zala, D.; Aebischer, P.; Déglon, N., 2002. Journal of Neuroscience. 3473 - 3483. 9. 22. 2002. http://www.scopus.com/inward/record.url?eid=2-s2.0-0036580677&partnerID=MN8TOARS . 10.1523/JNEUROSCI.3278-01.2002 . Journal of Neuroscience
Neuroprotective effect of interleukin-6 and IL6/IL6R chimera in the quinolinic acid rat model of Huntington's syndrome
Bensadoun, Jean-Charles; de Almeida, Luis Pereira; Dreano, Michel; Aebischer, Patrick; Deglon, Nicole; Bensadoun, J.-C.; Pereira de Almeida, L.; et al, 2001. European Journal of Neuroscience. 1753 - 1761. 11. 14. 2001. http://www.scopus.com/inward/record.url?eid=2-s2.0-0035783942&partnerID=MN8TOARS . 10.1046/j.0953-816x.2001.01802.x . published European Journal of Neuroscience
Neuroprotective Effect of a CNTF-Expressing Lentiviral Vector in the Quinolinic Acid Rat Model of Huntington's Disease
de Almeida, L.Pereira; Zala, D.; Aebischer, P.; Déglon, N., 2001. Neurobiology of Disease. 433 - 446. 3. 8. 2001. 10.1006/nbdi.2001.0388 . published Neurobiology of Disease
Self-Inactivating Lentiviral Vectors with Enhanced Transgene Expression as Potential Gene Transfer System in Parkinson's Disease
Deglon, Nicole; Tseng, Jack L.; Bensadoun, Jean-Charles; Zurn, Anne D.; Arsenijevic, Yvan; Almeida, Luis Pereira De; Zufferey, Romain; Trono, Didier; Aebischer, Patrick, 2000. Human Gene Therapy. 179 - 190. 1. 11. 2000. 10.1089/10430340050016256 . published Human Gene Therapy
Modeling dissolution of sparingly soluble multisized powders
Pereira de Almeida, Luis ; Simões, Sérgio; Brito, Paulo; Portugal, António; Figueiredo, Margarida, 1997. Journal of Pharmaceutical Sciences. 726 - 732. 6. 86. 1997. 10.1021/js960417w . published Journal of Pharmaceutical Sciences
Testing the applicability of classical diffusional models to polydisperse systems
Simöes, Sérgio; Pereira de Almeida, Luis; Figueiredo, Margarida, 1996. International Journal of Pharmaceutics. 169 - 176. 1-2. 139. 1996. 10.1016/0378-5173(96)04596-6 . published International Journal of Pharmaceutics
5 141 de 141 Publicações
ERDERA
2024-09-01
Engenharia de enzimas CRISPR/Cas13 direcionadas ao RNA para o Tratamento da Ataxia Espinocerebelosa do Tipo 3
2024-03-01
GeneT
2024-01-01
CInTech: Polo Tecnológico de Inovação, Translação e Industrialização de Medicamentos Injetáveis Complexos
2023-01-01
ARDAT
2020-11-01
5 6 de 6 Projectos I&D
Descoberta de biomarcadores da "clínica para o laboratório": a caminho de um ensaio clínico para a doença de Machado-Joseph
2023-03-01
MicroRNA-specific small molecule modifiers as a new and promising therapeutic approach for Machado-Joseph disease/spinocerebellar ataxia type 3 (MJD/SCA3)
2021-09-01
Edição genómica para tratamento da doença de Machado-Joseph por splicing alternativo
2022-01-01
(Less)PlasticBrain: Plastificantes Bio Substitutos – Impacto no Desenvolvimento do Cérebro Humano
2022-05-03
Entrega por via endovenosa direcionada para o cérebro do vetor AAV-PHPeB que codifica para a hidroxilase do colesterol CYP46A1 em um modelo de murganho de ataxia espinocerebelosa do tipo 3: uma estratégia terapêutica promissora não-invasiva
2020-03-01
5 7 de 7 Projectos Passados de I&D
Método de prognóstico e conceção de terapias genéticas específicas para a doença de Machado-Joseph
Sequências de oligonucleótidos optimizadas para o diagnóstico baseado em PCR da etiologia genética da Doença de Machado-Joseph (MJD), a expansão de trinucleótidos CAG no gene ATXN3: a) comprimento da expansão e b) polimorfismos de nucleótido único (SNPs) associados ao alelo mutante. Método para diagnosticar e prever a progressão da MJD e para conceber estratégias específicas de terapia génica. Otimização de oligonucleotídeos e sondas para medir a expressão do mRNA mutante na MJD para complementar o diagnóstico genético e avaliar o potencial de terapias génicas, como a edição ou silenciamento de genes.
Método de entrega alternativo e eficiente de sequências de silenciamento para o tratamento de doenças cerebrais
Primeiras vesículas extracelulares (VEs) como veículos terapêuticos direccionados para o cérebro contendo sequências silenciadoras baseadas em miRNAs para tratar a Doença de Machado-Joseph (DMJ) após administração intranasal diária. Vantagens: - Linha celular para produzir continuamente os EVs modificados - 5 vezes mais entrega devido a um tropismo específico para os neurónios Enriquecimento de 2,5 vezes das sequências de silenciamento em VEs devido à presença de ExoMotifs Administração não invasiva: Via intranasal Um método de administração alternativo e eficiente de miRNAs/sequências de silenciamento para tratar a DMJ e outras doenças cerebrais.
Terapia genética direccionada e minimamente invasiva para a Doença de Machado-Joseph
Técnicas de silenciamento de RNA (por exemplo, RNAi) visando polimorfismos de nucleotídeo único (SNPs) no mRNA da ataxina-3 em desequilíbrio de ligação com a expansão causadora da Doença de Machado-Joseph. Entrega por vias minimamente invasivas, incluindo a administração intra-cerebrospinal e intravenosa, utilizando vectores virais adeno-associados (AAVs).
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