Gene and Stem Cell Therapies for the Brain

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

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

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. http://dx.doi.org/10.1016/j.omtn.2020.01.010 . 10.1016/j.omtn.2020.01.010 . Molecular Therapy - Nucleic Acids

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

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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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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

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

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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

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

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

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

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

MicroRNA-specific small molecule modifiers as a new and promising therapeutic approach for Machado-Joseph disease/spinocerebellar ataxia type 3 (MJD/SCA3)

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

Role of microRNA deregulation in Machado-Joseph Disease: On the development of a microRNA-based therapeutic strategy

2018-07-06

Direct Conversion of Astrocytes into Cerebellar Neurons as an Innovative Treatment for Spinocerebellar Ataxia Type-3

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