Innovative Therapies

Vectors, Gene and Cell Therapy

Gene Therapy for Brain

Cancer and infection (vaccines)

Viral and Non-Viral vectors

Stem Cells

Overview

The Group of Vectors, Gene and Cell Therapy 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:

  • Establishment of models of brain disease;
  • Study of disease mechanisms;
  • Development of new molecular and stem cell, therapeutic and prophylactic approaches for brain disorders, particularly neurodegenerative diseases, notably Machado-Joseph's disease (MJD)/ spinocerebellar ataxia type 3, as well as brain tumours.

 

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:

  • Lentiviral and adeno-associated AAV viral vectors;
  • Exosomes and nanoparticles targeted to the brain repair and vaccination;
  • Gene editing with CRISPR-Cas9;
  • Induced pluripotent stem cells for disease modelling;
  • Neural Stem Cell and mesenchymal stromal cells transplantation for brain repair;
  • Biomarker discovery from patient fluids.

 

Team

Publications

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Information about journal articles, updated at 17-11-2024, 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

Laetitia S. Gaspar; Magda M. Santana; Carina Henriques; Maria M. Pinto; Teresa M. Ribeiro-Rodrigues; Henrique Girão; Rui Jorge Nobre; Luís Pereira de Almeida, 2020. Molecular Therapy - Methods & Clinical Development. 2020. https://doi.org/10.1016/j.omtm.2020.07.012 . 10.1016/j.omtm.2020.07.012 . Molecular Therapy - Methods & Clinical Development

Protocol for the Characterization of the CAG Tract and Flanking Polymorphisms in Machado-Joseph Disease: Impact on Diagnosis and Development of Gene-Based Therapies

Lopes, Sara Monteiro; Faro, Rosário; Lopes, Miguel Monteiro; Onofre, Isabel; Mendonça, Nuno; Ribeiro, Joana; Januário, Cristina; Nobre, Rui Jorge; Pereira de Almeida, Luís, 2020. The Journal of Molecular Diagnostics. 2020. http://dx.doi.org/10.1016/j.jmoldx.2020.03.003 . 10.1016/j.jmoldx.2020.03.003 . The Journal of Molecular Diagnostics

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

Lopes, C.; Tang, Y.; Anjo, S.I.; Manadas, B.; Onofre, I.; de Almeida, L.P.; Daley, G.Q.; Schlaeger, T.M.; Rego, A.C.C., 2020. Frontiers in Cell and Developmental Biology. 8. 2020. http://www.scopus.com/inward/record.url?eid=2-s2.0-85092059116&partnerID=MN8TOARS . 10.3389/fcell.2020.576592 . Frontiers in Cell and Developmental Biology

Mesenchymal Stromal Cells’ Therapy for Polyglutamine Disorders: Where Do We Stand and Where Should We Go?

Barros, I.; Marcelo, A.; Silva, T.P.; Barata, J.; Rufino-Ramos, D.; Pereira de Almeida, L.; Miranda, C.O., 2020. Frontiers in Cellular Neuroscience. 14. 2020. http://www.scopus.com/inward/record.url?eid=2-s2.0-85093862610&partnerID=MN8TOARS . 10.3389/fncel.2020.584277 . Frontiers in Cellular Neuroscience

Neurofilaments in spinocerebellar ataxia type 3: blood biomarkers at the preataxic and ataxic stage in humans and mice

Wilke, C.; Haas, E.; Reetz, K.; Faber, J.; Garcia-Moreno, H.; Santana, M.M.; van de Warrenburg, B.; et al, 2020. EMBO Molecular Medicine. 7. 12. 2020. http://www.scopus.com/inward/record.url?eid=2-s2.0-85086096828&partnerID=MN8TOARS . 10.15252/emmm.201911803 . EMBO Molecular Medicine

Trehalose alleviates the phenotype of Machado–Joseph disease mouse models

Santana, Magda M.; Paixão, Susana; Cunha-Santos, Janete; Silva, Teresa Pereira; Trevino-Garcia, Allyson; Gaspar, Laetitia S.; Nóbrega, Clévio; et al, 2020. Journal of Translational Medicine. 1. 18. 2020. http://dx.doi.org/10.1186/s12967-020-02302-2 . 10.1186/s12967-020-02302-2 . Journal of Translational Medicine

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

Lopes, Carla; Schlaeger, Thorsten; Pereira de Almeida, Luis ; Daley, George Q.; Ana Cristina Carvalho Rego, 2019. European Journal of Clinical Investigation. 53 - 100. S1. 49. 2019. http://dx.doi.org/10.1111/eci.13108 . 10.1111/eci.13108 . European Journal of Clinical Investigation

Restoring brain cholesterol turnover improves autophagy and has therapeutic potential in mouse models of spinocerebellar ataxia

Nóbrega, Clévio; Mendonca, Liliana; Marcelo, Adriana; Lamaziere, Antonin; Tome, Sandra; Despres, Gaetan; Matos, Carlos A; et al, 2019. Acta Neuropathologica. 2019. http://hdl.handle.net/10400.1/14197 . 10.1007/s00401-019-02019-7 . Acta Neuropathologica

Neural Stem Cells of Parkinson's Disease Patients Exhibit Aberrant Mitochondrial Morphology and Functionality.

Almeida, Luis, 2019. Stem cell reports. 2019. http://europepmc.org/articles/PMC6522948 . 10.1016/j.stemcr.2019.03.004 . Stem cell reports

RNA interference therapy for Machado-Joseph disease: Long-term safety profile of lentiviral vectors encoding short hairpin RNAs targeting mutant ataxin-3

Nóbrega, Clévio; Codesso, Jose Miguel; Mendonca, Liliana; de Almeida, Luis Pereira, 2019. 2019. http://hdl.handle.net/10400.1/14340 . 10.1089/hum.2018.157 .

Antisense oligonucleotide therapeutics in neurodegenerative diseases: the case of polyglutamine disorders

Ana C Silva; Diana D Lobo; Inês M Martins; Sara M Lopes; Carina Henriques; Sónia P Duarte; Jean-Cosme Dodart; Rui Jorge Nobre; Luis Pereira de Almeida, 2019. Brain. 2019. https://doi.org/10.1093/brain/awz328 . 10.1093/brain/awz328 . Brain

Successes and Hurdles in Stem Cells Application and Production for Brain Transplantation

Henriques, Daniel; Moreira, Ricardo; Schwamborn, Jens; Pereira de Almeida, Luís; Mendonça, Liliana S., 2019. Frontiers in Neuroscience. 13. 2019. http://dx.doi.org/10.3389/fnins.2019.01194 . 10.3389/fnins.2019.01194 . Frontiers in Neuroscience

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

Oliveira Miranda, C.; Marcelo, A.; Silva, T.P.; Barata, J.; Vasconcelos-Ferreira, A.; Pereira, D.; Nóbrega, C.; et al, 2018. Molecular Therapy. 2018. http://www.scopus.com/inward/record.url?eid=2-s2.0-85050950218&partnerID=MN8TOARS . 10.1016/j.ymthe.2018.07.007 . Molecular Therapy

Loss of hierarchical imprinting regulation at the Prader-Willi/Angelman syndrome locus in human iPSCs

Pólvora-Brandão, D.; Joaquim, M.; Godinho, I.; Aprile, D.; Álvaro, A.R.; Onofre, I.; Raposo, A.C.; et al, 2018. Human molecular genetics. 3999 - 4011. 23. 27. 2018. http://www.scopus.com/inward/record.url?eid=2-s2.0-85056694837&partnerID=MN8TOARS . 10.1093/hmg/ddy274 . Human molecular genetics

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

Rodrigues, C.F.D.; Serrano, E.; Patrício, M.I.; Val, M.M.; Albuquerque, P.; Fonseca, J.; Gomes, C.M.F.; et al, 2018. Scientific Reports. 1. 8. 2018. http://www.scopus.com/inward/record.url?eid=2-s2.0-85050990420&partnerID=MN8TOARS . 10.1038/s41598-018-29947-w . Scientific Reports

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

Cristo, F.; Inácio, J.M.; Rosas, G.; Carreira, I.M.; Melo, J.B.; de Almeida, L.P.; Mendes, P.; et al, 2017. Stem Cell Research. 152 - 156. 25. 2017. http://www.scopus.com/inward/record.url?eid=2-s2.0-85033403571&partnerID=MN8TOARS . 10.1016/j.scr.2017.10.019 . Stem Cell Research

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

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