Innovative Therapies

Gene and Stem Cell Therapies for the Brain

Gene Therapy

Viral Vectors

Stem Cells

Neurodegenerative Diseases

Machado-Joseph Disease

Research lines

Models of disease

Mechanism driven therapies

Stem cell transplantation

Gene silencing

Disease biomarkers research in patients



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Information about journal articles, updated at 17-12-2023, from platform CIÊNCIAVITAE.

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. . 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. . 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. . 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. . 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. . 10.3390/ijms25031584 . International Journal of Molecular Sciences

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. . 10.1016/j.ymthe.2023.05.012 . in press Molecular Therapy

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. . 10.1530/JME-21-0272 . under revision Journal of Molecular Endocrinology

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. . 10.3390/biom13020278 . Biomolecules

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. . 10.1007/s12311-023-01547-z . 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. . 10.1016/j.ymthe.2023.04.001 . Molecular Therapy

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. . 10.1007/s12311-023-01546-0 . Cerebellum

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. . 10.1111/nan.12892 . Neuropathology and Applied Neurobiology

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. . 10.3390/polym15102256 . Polymers

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. . 10.1093/brain/awad128 . 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. . 10.1111/acel.13983 . Aging Cell

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. . 10.1093/brain/awac473 . Brain

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. . 10.1007/s43630-023-00476-4 . Photochemical and Photobiological Sciences

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. . 10.3390/ijms241612603 . International Journal of Molecular Sciences

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. . 10.1007/s00415-023-12064-8 . Journal of Neurology

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. . 10.1007/s00415-022-11441-z . 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. . 10.1111/nan.12748 . Neuropathology and Applied Neurobiology

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. . 10.1002/mds.28844 . Movement Disorders

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. . 10.1016/j.ymthe.2021.07.012 . Molecular Therapy

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. . 10.1089/nat.2021.0020 . Nucleic Acid Therapeutics

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. . 10.1111/nan.12763 . Neuropathology and Applied Neurobiology

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. . 10.1186/s40478-022-01329-4 . Acta Neuropathologica Communications

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. . 10.1007/s43630-022-00202-6 . Photochemical and Photobiological Sciences

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. . 10.1007/s40520-022-02257-y . Aging Clinical and Experimental Research

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. . 10.1038/s41434-022-00372-z . Gene 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. . 10.1016/j.biomaterials.2022.121366 . Biomaterials

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. . 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. . 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. . 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. . 10.1002/smll.202203999 . Small

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. . 10.1002/mds.29135 . Movement Disorders

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

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. . 10.1038/s41598-022-16369-y . Scientific Reports

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. . 10.3389/fbioe.2022.1082883 . Frontiers in Bioengineering and Biotechnology

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. . 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. . 10.1080/15548627.2020.1797280 . Autophagy

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. . 10.3390/ijms22147294 . 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. . 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. . 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. . 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. . 10.3390/pharmaceutics13101598 . Pharmaceutics

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. . 10.3390/ijms222212288 . International Journal of Molecular Sciences

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. . 10.1038/s41419-021-03873-8 . Cell Death and Disease

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. . 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. . 10.1038/s41598-021-82339-5 . Scientific Reports

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. . 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

Barros-Viegas, Ana Teresa; Carmona, Vítor; Ferreiro, Elisabete; Guedes, Joana; Cardoso, Ana Maria; Cunha, Pedro; Pereira de Almeida, Luís; et al, 2020. Molecular Therapy - Nucleic Acids. 1219 - 1236. 19. 2020. . 10.1016/j.omtn.2020.01.010 . Molecular Therapy - Nucleic Acids

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. . 10.1186/s12967-020-02302-2 . Journal of Translational Medicine

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. . 10.1093/gerona/glz280 . The Journals of Gerontology: Series A

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. . 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. . 10.1016/j.omtm.2020.07.012 . Molecular Therapy - Methods & Clinical Development

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. . 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. . 10.3389/fncel.2020.584277 . Frontiers in Cellular Neuroscience

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. 10.1016/j.omtm.2020.07.012 . published Molecular Therapy - Methods & Clinical Development

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. . 10.15252/emmm.201911803 . EMBO Molecular Medicine

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. . 10.1007/s00401-019-02019-7 . Acta Neuropathologica

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. . 10.1093/brain/awz328 . Brain

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. . 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. . 10.1093/hmg/ddz097 . Human Molecular Genetics

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. . 10.1089/hum.2018.157 .

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

Almeida, Luis, 2019. Stem cell reports. 2019. . 10.1016/j.stemcr.2019.03.004 . Stem cell reports

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. . 10.3389/fnins.2019.01194 . Frontiers in Neuroscience

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. . 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. . 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. . 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. . 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. . 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. . 10.1016/j.scr.2017.10.019 . Stem Cell Research

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. . 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. . 10.2174/1381612822666161227121912 . Current Pharmaceutical Design

Extracellular vesicles: Novel promising delivery systems for therapy of brain diseases

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