The group is devoted to the creation of new effective therapies for brain diseases that can be moved from the bench to the bedside. For this, the group designs and uses technological platforms based on viral and non-viral gene transfer vectors, and induced pluripotent stem cells for the:
The group results from a restructuration of the former “Vectors and Gene Therapy” group, and has a strong expertise in gene transfer (viral and non-viral) and stem cells for brain disease-modeling and therapy. Our technological platforms include:
Information about journal articles, updated at 22-12-2024, from platform 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
Diana Duarte Lobo; Rui Jorge Nobre; Catarina Oliveira Miranda; Dina Pereira; João Castelhano; José Sereno; Arnulf Koeppen; Miguel Castelo-Branco; Luís Pereira de Almeida, 2020. Acta Neuropathologica Communications. 2020. https://doi.org/10.1186/s40478-020-00955-0 . 10.1186/s40478-020-00955-0 . Acta Neuropathologica Communications
Simple and Fast SEC-Based Protocol to Isolate Human Plasma-Derived Extracellular Vesicles for Transcriptional Research
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Mesenchymal Stromal Cells’ Therapy for Polyglutamine Disorders: Where Do We Stand and Where Should We Go?
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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
Marques, A.P.; Cunha-Santos, J.; Leal, H.; Sousa-Ferreira, L.; Pereira de Almeida, L.; Cavadas, C.; Rosmaninho-Salgado, J., 2018. Biochimica et Biophysica Acta - General Subjects. 403 - 413. 3. 1862. 2018. http://www.scopus.com/inward/record.url?eid=2-s2.0-85037668547&partnerID=MN8TOARS . 10.1016/j.bbagen.2017.11.012 . Biochimica et Biophysica Acta - General Subjects
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
Hachigian, L.J.; Carmona, V.; Fenster, R.J.; Kulicke, R.; Heilbut, A.; Sittler, A.; Pereira de Almeida, L.; et al, 2017. Cell Reports. 2688 - 2695. 10. 21. 2017. http://www.scopus.com/inward/record.url?eid=2-s2.0-85038085324&partnerID=MN8TOARS . 10.1016/j.celrep.2017.11.018 . Cell Reports
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
Rufino-Ramos, D.; Albuquerque, P.R.; Carmona, V.; Perfeito, R.; Nobre, R.J.; Pereira de Almeida, L., 2017. Journal of Controlled Release. 247 - 258. 262. 2017. http://www.scopus.com/inward/record.url?eid=2-s2.0-85026758579&partnerID=MN8TOARS . 10.1016/j.jconrel.2017.07.001 . Journal of Controlled Release
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 out of 141 Publications
5 6 out of 6 R&D Projects
From “bedside-to-bench” biomarker discovery: on the road to a clinical trial for Machado-Joseph Disease
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
Genome editing to rescue Machado-Joseph disease by alternative splicing
2022-01-01
(Less)PlasticBrain: Bio Replacing Plasticizers – Impact in the Human Brain Development
2022-05-03
Intravenous delivery of the brain-targeting AAV-PHPeB encoding the cholesterol hydroxylase CYP46A1 into a mouse model of spinocerebellar ataxia type 3: a promising non-invasive therapeutic strategy
2020-03-01
5 7 out of 7 Past R&D Projects
Method for prognosis and design of specific gene therapies for Machado-Joseph Disease
Optimized oligonucleotide sequences for PCR-based diagnostics of the genetic aetiology of Machado-Joseph Disorder (MJD), the CAG trinucleotide expansion within ATXN3 gene: a) length of the expansion and b) single-nucleotide polymorphisms (SNPs) associated with the mutant allele. Method to diagnose and predict the progression of MJD and to design specific gene therapy strategies. Optimized oligonucleotides and probes to measure the expression of mutant mRNA in MJD to complement the genetic diagnosis and to evaluate the potential of gene-based therapies such as gene editing or silencing.
Alternative and efficient delivery method of silencing sequences for the treatment of brain diseases
First extracellular vesicles (EVs) as therapeutic vehicles targeting the brain containing engineered miRNA-based silencing sequences to treat Machado-Joseph Disease (MJD) upon daily intranasal administration. Advantages: Packaging cell line to continuously produce engineered EVs 5x more delivery due to a specific tropism for neurons 2.5-fold enrichment of silencing sequences into EVs due to the presence of ExoMotifs Non-invasive administration: Intranasal route An alternative and efficient delivery method for miRNAs/silencing sequences to treat MJD and other brain diseases
Targeted and minimal invasive gene therapy for Machado-Joseph Disease
RNA silencing techniques (e.g. RNAi) targeting single nucleotide polymorphisms (SNPs) in the ataxin-3 mRNA in linkage disequilibrium with the Machado Joseph disease-causing expansion. Delivery via minimally invasive routes, including intra-cerebrospinal and intravenous administration, using adeno-associated viral vectors (AAVs).
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