Novel Strategies for Cell-based Neural Reconstruction

CORDIS fournit des liens vers les livrables publics et les publications des projets HORIZON.

Les liens vers les livrables et les publications des projets du 7e PC, ainsi que les liens vers certains types de résultats spécifiques tels que les jeux de données et les logiciels, sont récupérés dynamiquement sur OpenAIRE .

Livrables

Intermediate assessment of the therapeutic efficacy and potency of stem cell-derived MSNs
Report on the generation of novel hPSC lines and virus engineered for cell replacement
To define the cytokine environment post grafting at the transplant site using microdialysis
Report on the protocols on next-generation DA, MSN and cortical neurons for cell replacement based on human gene function analysis , including concept for scale-up and GMP adaptation of selected protocol(s).

Report on the protocols on next-generation DA, MSN and cortical neurons for cell replacement based on human gene function analysis, including concept for scale-up and GMP adaptation of selected protocol(s).

Complete preclinical validation of optimised hPSC-derived MSNs
Extensive pre-clinical validation of new generation A9 DA neurons
Evaluation of graft enhancements for increased therapeutic effect, including designer grafts to support simple circuit replacement and activity regulation approaches
Definition of optimal graft composition
Complete profiling of optimized grafts using multiplexed molecular and protein-based methods
Report on the effects of host mHtt pathology on grafted MSNs
Report summarising the outcome of workshop on Transplant connectomics: strategies to improve the structural and functional integration of replacement cells in neurodegenerative conditions and trauma

Report summarising the outcome of workshop on Transplant connectomics strategies to improve the structural and functional integration of replacement cells in neurodegenerative conditions and trauma

To define the immunogenicity of new generation hPSC derived dopaminergic, MSN and cortical neurons in vitro and in vivo
Final Deliverable, Final report on the impact of communication, dissemination and training activities
Documentation of functional efficacy and circuitry integration of in situ converted DA neurons
First report on the effects of host mHtt pathology on grafted MSNs
Report on the ability of grafted programmed cells to undergo circuit integration in the adult rodent brain
First protocols for in vivo MSN reprogramming from glia
Report on best available tools for circuitry analysis and a list of priorities for future improvements
Intermediate preclinical validation of optimised hPSC-derived MSNs

Intermediate preclinical validation of optimised hPSCderived MSNs

Evaluation of graft enhancements for increased therapeutic effect, including non-DA dependent aspects
Report summarising the outcome of workshop on Regulatory and ethical issues related to the development and clinical use of next generation cell therapy products
A comprehensive definition of endogenous human cortical, striatal and midbrain cell-types and their gene expression profiles

A comprehensive definition of endogenous human cortical striatal and midbrain celltypes and their gene expression profiles

Report on the protocols and next-generation hPSC-derived or converted DA, MSN, cortical, telencephalic interneurons and basal forebrain cholinergic neurons for cell replacement based on human gene expression profiles
Report on graft composition of the current state of the art transplants based on new methodologies developed within the WP
To make defined MHC knock out hPSC derived neurons and study in animal models of disease
Report on the efficiency and specificity of inducing cortico-striatal projection neurons from local glia in vivo
Report on the function of TFs specifically expressed in human cortical, MSN and mDA neurons
Protocols for comprehensive image-based assessment of graft-host connectomes and donor cell function

Protocols for comprehensive imagebased assessment of grafthost connectomes and donor cell function

Complete assessment of the therapeutic efficacy and potency of stem cell-derived MSNs
Report on the ability of grafted human neurons to integrate and restore visual and motor dysfunction
Intermediate report on the impact of communication, dissemination and training activities

Intermediate report on the impact of communication dissemination and training activities

Report summarising the outcome of workshop on Strategies and goals for the development of next generation cell therapy products for cell replacement in brain disorders
Report summarising the outcome of workshop on Strategies for the development of therapeutic cell products that can evade the host´s immune system, including the development of tools to monitor graft rejection in clinical trials

NSC-Reconstruct dedicated website is online

NSCReconstruct dedicated website is online

Description and Implementation of the Data Management Plan
ORDP: Open Research Data Pilot Defined

ORDP Open Research Data Pilot Defined

Publications

Protocol for optical clearing and imaging of fluorescently labeled ex vivo rat brain slices

Auteurs: Giacomoni J, Habekost M, Cepeda-Prado E, Mattsson B, Ottosson DR, Parmar M, Kajtez J
Publié dans: STAR Protoc, Numéro Mar 17;4(1):102041, 2023, ISSN 2666-1667
Éditeur: Cell Press
DOI: 10.1016/j.xpro.2022.102041

A protocol for the differentiation of human embryonic stem cells into midbrain dopaminergic neurons

Auteurs: Nishimura K, Ásgrímsdóttir ES, Yang S, Arenas E
Publié dans: STAR Protocols, Numéro Volume 4, Numéro 3, 15 September 2023, 2023, ISSN 2666-1667
Éditeur: Cell Press Elsevier Inc
DOI: 10.1016/j.xpro.2023.102355

Spatial Centrosome Proteomic Profiling of Human iPSC-derived Neural Cells

Auteurs: Fatma Uzbas, Adam O’Neill
Publié dans: BIO-PROTOCOL, Numéro 13, 2024, ISSN 2331-8325
Éditeur: Bio-protocol LLC
DOI: 10.21769/bioprotoc.4812

Dynamic spatiotemporal activation of a pervasive neurogenic competence in striatal astrocytes supports continuous neurogenesis following injury

Auteurs: Marco Fogli, Giulia Nato, Philip Greulich, Jacopo Pinto, Marta Ribodino, Gregorio Valsania, Paolo Peretto, Annalisa Buffo, Federico Luzzati
Publié dans: Stem Cell Reports, Numéro 19, 2024, Page(s) 1432-1450, ISSN 2213-6711
Éditeur: Cell Press
DOI: 10.1016/j.stemcr.2024.08.006

Single cell transcriptomics reveals correct developmental dynamics and high-quality midbrain cell types by improved hESC differentiation

Auteurs: Kaneyasu Nishimura, Shanzheng Yang, Ka Wai Lee, Emilia Sif Asgrimsdottir, Kasra Nikodei, Wojciech Paslawski, Gnodde Sabine, Guochang Lyu, Lijuan Hu, Carmen Saltó, Per Svenningsson, Jens Hjerling-Leffler, Sten Linnarsson, Ernest Arenas
Publié dans: Stem Cell Reports, Numéro Vol. 18 Numéro 1, 2023, Page(s) 337-353, ISSN 2213-6711
Éditeur: Cell Press
DOI: 10.1016/j.stemcr.2022.10.016

LRRC37B is a human modifier of voltage-gated sodium channels and axon excitability in cortical neurons

Auteurs: Libé-Philippot B, Lejeune A, Wierda K, Vlaeminck I, Beckers S, Gaspariunaite V, Bilheu A, Nyitrai H, Vennekens KM, Bird TW, Soto D, Dennis MY, Comoletti D, Theys T, de Wit J, Vanderhaeghen P
Publié dans: Cell, Numéro 186(26), 2023, Page(s) 5766-5783.e25, ISSN 1097-4172
Éditeur: Cell Press
DOI: 10.1016/j.cell.2023.11.028

Excessive local host-graft connectivity in aging and amyloid-loaded brain

Auteurs: Thomas J, Martinez-Reza MF, Thorwirth M, Zarb Y, Conzelmann KK, Hauck SM, Grade S, Götz M
Publié dans: Science Advances, Numéro 10;8(23), 2022, Page(s) eabg9287, ISSN 2375-2548
Éditeur: American Association for the Advancement of Science
DOI: 10.1126/sciadv.abg9287

Probing cell identity hierarchies by fate titration and collision during direct reprogramming

Auteurs: Bob A Hersbach, David S Fischer, Giacomo Masserdotti, Deeksha, Karolina Mojžišová, Thomas Waltzhöni, Diego Rodriguez-Terrones, Matthias Heinig, Fabian J Theis, Magdalena Götz, Stefan H Stricker
Publié dans: Mol Syst Biol., 2022, Page(s) e11129, ISSN 1744-4292
Éditeur: Nature Publishing Group
DOI: 10.15252/msb.202211129

Generation of human cerebral organoids with a structured outer subventricular zone

Auteurs: Walsh RM, Luongo R, Giacomelli E, Ciceri G, Rittenhouse C, Verrillo A, Galimberti M, Bocchi VD, Wu Y, Xu N, Mosole S, Muller J, Vezzoli E, Jungverdorben J, Zhou T, Barker RA, Cattaneo E, Studer L, Baggiolini A
Publié dans: Cell Reports, Numéro 43(4), 2024, Page(s) 114031, ISSN 2211-1247
Éditeur: Cell Press
DOI: 10.1016/j.celrep.2024.114031

Stem cell programming – prospects for perinatal medicine

Auteurs: Berg, L.J., Brüstle, O
Publié dans: Journal of Perinatal Medicine, Numéro 2023 Feb 22;51(6), 2023, Page(s) 730-736, ISSN 0300-5577
Éditeur: Walter de Gruyter GmbH & Co. KG
DOI: 10.1515/jpm-2022-0575

Generation of Human Ventral Midbrain Organoids Derived from Pluripotent Stem Cells

Auteurs: Edoardo Sozzi, Fredrik Nilsson, Janko Kajtez, Malin Parmar, Alessandro Fiorenzano
Publié dans: Current protocols., 2022, ISSN 2691-1299
Éditeur: Hoboken, NJ : John Wiley & Sons, [2021]-
DOI: 10.1002/cpz1.555

Heterogeneity of neurons reprogrammed from spinal cord astrocytes by the proneural factors Ascl1 and Neurogenin2

Auteurs: J. Kempf, K. Knelles, B.A. Hersbach, D. Petrik, T. Riedemann, V. Bednarova, A. Janjic, T. Simon-Ebert, W. Enard, P. Smialowski, M. Götz, G. Masserdotti
Publié dans: Cell Reports, Numéro 36/3, 2021, Page(s) 109409, ISSN 2211-1247
Éditeur: Cell Press
DOI: 10.1016/j.celrep.2021.109409

Injury-specific factors in the cerebrospinal fluid regulate astrocyte plasticity in the human brain

Auteurs: Swetlana Sirko, Christian Schichor, Patrizia Della Vecchia, Fabian Metzger, Giovanna Sonsalla, Tatiana Simon, Martina Bürkle, Sofia Kalpazidou, Jovica Ninkovic, Giacomo Masserdotti, Jean-Frederic Sauniere, Valentina Iacobelli, Stefano Iacobelli, Claire Delbridge, Stefanie M. Hauck, Jörg-Christian Tonn & Magdalena Götz
Publié dans: Nature Medicine, Numéro 29, 2023, Page(s) 149–3161, ISSN 1546-170X
Éditeur: Nature Publishing Group
DOI: 10.1038/s41591-023-02644-6

A novel two-factor monosynaptic TRIO tracing method for assessment of circuit integration of hESC-derived dopamine transplants

Auteurs: Aldrin-Kirk P, Åkerblom M, Cardoso T, Nolbrant S, Adler AF, Liu X, Heuer A, Davidsson M, Parmar M, Björklund T
Publié dans: Stem Cell Reports, 2022, ISSN 2213-6711
Éditeur: Cell Press
DOI: 10.1016/j.stemcr.2021.11.014

Patterning effects of FGF17 and cAMP on generation of dopaminergic progenitors for cell replacement therapy in Parkinson’s disease

Auteurs: Amalie Holm Nygaard, Alrik L Schörling, Zehra Abay-Nørgaard, Erno Hänninen, Yuan Li, Adrian Santonja, Gaurav Singh Rathore, Alison Salvador, Charlotte Rusimbi, Katrine Bech Lauritzen, Yu Zhang, Agnete Kirkeby
Publié dans: Stem Cells, 2025, ISSN 1066-5099
Éditeur: AlphaMed Press Inc
DOI: 10.1093/stmcls/sxaf004

Transcriptomic diversity of cell types across the adult human brain

Auteurs: Kimberly Siletti*, Rebecca Hodge†, Alejandro Mossi Albiach†, Ka Wai Lee, Song-Lin Ding Lijuan Hu, Peter Lönnerberg, Trygve Bakken, Tamara Casper, Michael Clark, Nick Dee, Jessica Gloe, Daniel Hirschstein , Nadiya V Shapovalova, C. Dirk Keene, Julie Nyhus, Herman Tung, Anna Marie Yanny, Ernest Arenas, Ed S. Lein, Sten Linnarsson
Publié dans: Science, Numéro Vol 382, Numéro 6667, 13 October 2023, 2023, ISSN 0036-8075
Éditeur: American Association for the Advancement of Science
DOI: 10.1126/science.add7046

A Novel LHX6 Reporter Cell Line for Tracking Human iPSC-Derived Cortical Interneurons

Auteurs: Maria Cruz-Santos, Lucia Fernandez Cardo, Meng Li
Publié dans: Cells, Numéro 11(5), 853, 2022, ISSN 2073-4409
Éditeur: MDPI
DOI: 10.3390/cells11050853

Silk scaffolding drives self-assembly of functional and mature human brain organoids

Auteurs: Edoardo Sozzi, Janko Kajtez, Andreas Bruzelius, Milan Finn Wesseler, Fredrik Nilsson, Marcella Birtele, Niels B. Larsen, Daniella Rylander Ottosson, Petter Storm, Malin Parmar, Alessandro Fiorenzano
Publié dans: Frontiers in cell and developmental biology, 2022, ISSN 2296-634X
Éditeur: Lausanne : Frontiers Media S.A., [2013]-
DOI: 10.3389/fcell.2022.1023279

A defined human-specific platform for modeling neuronal network stimulation in vitro and in silico

Auteurs: Jianbin Wen, Michael Peitz, Oliver Brüstle
Publié dans: Journal of Neuroscience Methods, Numéro 373, 2022, ISSN 0165-0270
Éditeur: Elsevier BV
DOI: 10.1016/j.jneumeth.2022.109562

Midbrain Dopaminergic Neuron Development at the Single Cell Level: In vivo and in Stem Cells

Auteurs: Emilía Sif Ásgrímsdóttir, Ernest Arenas
Publié dans: Frontiers in Cell and Developmental Biology, Numéro 8, 2020, ISSN 2296-634X
Éditeur: Frontier Media SA
DOI: 10.3389/fcell.2020.00463

In vivo conversion of dopamine neurons in mouse models of Parkinson’s disease — a future approach for regenerative therapy?

Auteurs: Malin Parmar, Anders Björklund, Tomas Björklund
Publié dans: Current Opinion in Genetics & Development, 2021, Page(s) 76-82, ISSN 0959-437X
Éditeur: Elsevier BV
DOI: 10.1016/j.gde.2021.06.002

Single Cell Deletion of the Transcription Factors Trps1 and Sox9 in Astrocytes Reveals Novel Functions in the Adult Cerebral Cortex

Auteurs: Poornemaa Natarajan, Christina Koupourtidou, Thibault de Resseguier, Manja Thorwirth, Riccardo Bocchi, Judith Fischer‐Sternjak, Sarah Gleiss, Diana Rodrigues, Michael H. Myoga, Jovica Ninkovic, Giacomo Masserdotti, Magdalena Götz
Publié dans: Glia, 2024, ISSN 0894-1491
Éditeur: John Wiley & Sons Inc.
DOI: 10.1002/glia.24645

Brain injury environment critically influences the connectivity of transplanted neurons

Auteurs: Sofia Grade, Judith Thomas, Yvette Zarb, Manja Thorwirth, Karl-Klaus Conzelmann, Stefanie M. Hauck, Magdalena Götz
Publié dans: Science Advances, Numéro 8, 2024, ISSN 2375-2548
Éditeur: AAAS
DOI: 10.1126/sciadv.abg9445

Imaging three-dimensional brain organoid architecture from meso- to nanoscale across development

Auteurs: Juan Eduardo Rodriguez-Gatica; Vira Iefremova; Liubov Sokhranyaeva; Si Wah Christina Au Yeung; Yannik Breitkreuz; Oliver Brüstle; Martin Karl Schwarz; Ulrich Kubitscheck
Publié dans: Development, Numéro 149, 2022, ISSN 0950-1991
Éditeur: The Company of Biologists Ltd.
DOI: 10.1242/dev.200439

Direct neuronal reprogramming: Fast forward from new concepts toward therapeutic approaches

Auteurs: Riccardo Bocchi, Giacomo Masserdotti, Magdalena Götz
Publié dans: Neuron, Numéro 110(3), 2022, Page(s) 366-393, ISSN 0896-6273
Éditeur: Cell Press
DOI: 10.1016/j.neuron.2021.11.023

ADHD-associated PARK2 copy number variants: A pilot study on gene expression and effects of supplementary deprivation in patient-derived cell lines

Auteurs: Franzsika Radtke,Viola Stella Palladino,Rhiannon V McNeill, Andreas G Chiocchetti, Denise Haslinger, Matthias Leyh, Danijel Gersic, Markus Frank, Lena Grünewald, Stephan Klebe, Oliver Brüstle, Katharina Günther, Frank Edenhofer, Thorsten M Kranz, Andreas Reif,Sarah Kittel-Schneider
Publié dans: American Journal of Medical Genetics Part B: Neuropsychiatric Genetics, Numéro 189(7-8), 2022, ISSN 1552-4876
Éditeur: Wiley Periodicals LLC.
DOI: 10.1002/ajmg.b.32918

Identification of TGFβ signaling as a regulator of interneuron neurogenesis in a human pluripotent stem cell model

Auteurs: Maria Cruz Santos, Meng Li
Publié dans: Neuronal Signaling, Numéro 5 (4), 2021, ISSN 2059-6553
Éditeur: Portland Press
DOI: 10.1042/ns20210020

Clinical translation of pluripotent stem cell-based therapies: successes and challenges

Auteurs: Josefine Rågård Christiansen, Agnete Kirkeby
Publié dans: Development, Numéro 151, 2024, ISSN 0950-1991
Éditeur: The Company of Biologists Ltd.
DOI: 10.1242/dev.202067

SYNGAP1 deficiency disrupts synaptic neoteny in xenotransplanted human cortical neurons in vivo

Auteurs: Ben Vermaercke, Ryohei Iwata, Keimpe Wierda, Leïla Boubakar, Paula Rodriguez, Martyna Ditkowska, Vincent Bonin, Pierre Vanderhaeghen
Publié dans: Neuron, Numéro 112, 2024, Page(s) 3058-3068.e8, ISSN 0896-6273
Éditeur: Cell Press
DOI: 10.1016/j.neuron.2024.07.007

Single-cell transcriptomics captures features of human midbrain development and dopamine neuron diversity in brain organoids

Auteurs: Alessandro Fiorenzano, Edoardo Sozzi, Marcella Birtele, Janko Kajtez, Jessica Giacomoni, Fredrik Nilsson, Andreas Bruzelius, Yogita Sharma, Yu Zhang, Bengt Mattsson, Jenny Emnéus, Daniella Rylander Ottosson, Petter Storm, Malin Parmar
Publié dans: Nat Commun, 2021, ISSN 2041-1723
Éditeur: Nature Publishing Group
DOI: 10.1038/s41467-021-27464-5

Generation of a fluorescent hESC reporter line (Kle033-A-1) for the isolation of distinct midbrain progenitor cell types

Auteurs: Emilía Sif Ásgrímsdóttir, Ernest Arenas
Publié dans: Stem Cell Research, Numéro 81, 2025, Page(s) 103523, ISSN 1873-5061
Éditeur: Elsevier
DOI: 10.1016/j.scr.2024.103523

Direct neuronal reprogramming of NDUFS4 patient cells identifies the unfolded protein response as a novel general reprogramming hurdle

Auteurs: Giovanna Sonsalla, Ana Belen Malpartida, Therese Riedemann, Mirjana Gusic, Ejona Rusha, Giorgia Bulli, Sonia Najas, Aleks Janjic, Bob A. Hersbach, Pawel Smialowski, Micha Drukker, Wolfgang Enard, Jochen H.M. Prehn, Holger Prokisch, Magdalena Götz, Giacomo Masserdotti
Publié dans: Neuron, Numéro 112, 2024, Page(s) 1117-1132.e9, ISSN 0896-6273
Éditeur: Cell Press
DOI: 10.1016/j.neuron.2023.12.020

Comprehensive cell atlas of the first-trimester developing human brain

Auteurs: Emelie Braun, Miri Danan-Gotthold, Lars E. Borm, Elin Vinsland, Ka Wai Lee, Peter Lönnerberg, Lijuan Hu, Xiaofei Li, Xiaoling He, Žaneta Andrusivová, Joakim Lundeberg, Roger A. Barker, Ernest Arenas, Erik Sundström, Sten Linnarsson
Publié dans: Science, Numéro Vol 382, Numéro 6667, 13 October 2023, 2023, ISSN 0036-8075
Éditeur: American Association for the Advancement of Science
DOI: 10.1126/science.adf1226

Combinatorial ECM arrays identify cooperative roles for matricellular proteins in enhancing the generation of TH+ neurons from human pluripotent cells

Auteurs: Maqsood Ahmed, Matthew J. Owens, Enrique M. Toledo, Ernest Arenas, Mark Bradley, Charles ffrench-Constant
Publié dans: Frontiers in Cell and Developmental Biology, Numéro Volume 9 - 2021, 2021, ISSN 2296-634X
Éditeur: Lausanne : Frontiers Media S.A., [2013]-
DOI: 10.3389/fcell.2021.755406

Comparing Viral Vectors and Fate Mapping Approaches for Astrocyte-to-Neuron Reprogramming in the Injured Mouse Cerebral Cortex

Auteurs: Matteo Puglisi, Chu Lan Lao, Gulzar Wani, Giacomo Masserdotti, Riccardo Bocchi, Magdalena Götz
Publié dans: Cells, Numéro 13, 2024, Page(s) 1408, ISSN 2073-4409
Éditeur: MDPI
DOI: 10.3390/cells13171408

Synaptic neoteny of human cortical neurons requires species-specific balancing of SRGAP2-SYNGAP1 cross-inhibition

Auteurs: Baptiste Libé-Philippot, Ryohei Iwata, Aleksandra J. Recupero, Keimpe Wierda, Sergio Bernal Garcia, Luke Hammond, Anja van Benthem, Ridha Limame, Martyna Ditkowska, Sofie Beckers, Vaiva Gaspariunaite, Eugénie Peze-Heidsieck, Daan Remans, Cécile Charrier, Tom Theys, Franck Polleux, Pierre Vanderhaeghen
Publié dans: Neuron, Numéro 112, 2024, Page(s) 3602-3617.e9, ISSN 0896-6273
Éditeur: Cell Press
DOI: 10.1016/j.neuron.2024.08.021

Lineage tracing of stem cell–derived dopamine grafts in a Parkinson’s model reveals shared origin of all graft-derived cells

Auteurs: Petter Storm, Yu Zhang, Fredrik Nilsson, Alessandro Fiorenzano, Niklas Krausse, Malin Åkerblom, Marcus Davidsson, Joan Yuan, Agnete Kirkeby, Tomas Björklund, Malin Parmar
Publié dans: Science Advances, Numéro 10, 2024, ISSN 2375-2548
Éditeur: AAAS
DOI: 10.1126/sciadv.adn3057

Astrocyte heterogeneity reveals region-specific astrogenesis in the white matter

Auteurs: Riccardo Bocchi, Manja Thorwirth, Tatiana Simon-Ebert, Christina Koupourtidou, Solène Clavreul, Keegan Kolf, Patrizia Della Vecchia, Sara Bottes, Sebastian Jessberger, Jiafeng Zhou, Gulzar Wani, Gregor-Alexander Pilz, Jovica Ninkovic, Annalisa Buffo, Swetlana Sirko, Magdalena Götz, Judith Fischer-Sternjak
Publié dans: Nature Neuroscience, 2025, ISSN 1097-6256
Éditeur: Nature Publishing Group
DOI: 10.1038/s41593-025-01878-6

Preclinical quality, safety, and efficacy of a human embryonic stem cell-derived product for the treatment of Parkinson’s disease, STEM-PD

Auteurs: Agnete Kirkeby, Jenny Nelander, Deirdre B. Hoban, Nina Rogelius, Hjálmar Bjartmarz, Novo Nordisk Cell Therapy R&D, Petter Storm, Alessandro Fiorenzano, Andrew F. Adler, Shelby Vale, Janitha Mudannayake, Yu Zhang, Tiago Cardoso , Bengt Mattsson, Anne M. Landau, Andreas N Glud, Jens C. Sørensen, Thea P. Lillethorup, Mark Lowdell, Carla Carvalho, Owen Bain, Trinette van Vliet, Olle Lindvall, Anders
Publié dans: Cell Stem Cell, 2023, ISSN 1934-5909
Éditeur: Cell Press
DOI: 10.1016/j.stem.2023.08.014

Forced LMX1A expression induces dorsal neural fates and disrupts patterning of human embryonic stem cells into ventral midbrain dopaminergic neurons

Auteurs: Pedro Rifes, Janko Kajtez, Josefine Rågård Christiansen, Alrik Schörling, Gaurav Singh Rathore, Daniel A. Wolf, Andreas Heuer, Agnete Kirkeby
Publié dans: Stem Cell Reports, Numéro Volume 19, Numéro 6, 2024, Page(s) 830-838, ISSN 2213-6711
Éditeur: Cell Press
DOI: 10.1016/j.stemcr.2024.04.010

Identification of adeno-associated virus variants for gene transfer into human neural cell types by parallel capsid screening

Auteurs: Lea Jessica Flitsch; Kathleen Börner; Christian Stüllein; Simon Ziegler; Vera Sonntag-Buck; Ellen Wiedtke; Vesselina Semkova; Si Wah Christina Au Yeung; Julia Schlee; Mohamad Hajo; Mona Mathews; Beatrice Stefanie Ludwig; Susanne Kossatz; Horst Kessler; Dirk Grimm; Oliver Brüstle
Publié dans: Scientific Report, Numéro 12(1), 2022, ISSN 2045-2322
Éditeur: Nature Publishing Group
DOI: 10.1038/s41598-022-12404-0

Energy Metabolism Disturbances in Cell Models of PARK2 CNV Carriers with ADHD

Auteurs: Viola Stella Palladino, Andreas G. Chiocchetti, Lukas Frank, Denise Haslinger, Rhiannon McNeill, Franziska Radtke, Andreas Till, Simone Haupt, Oliver Brüstle, Katharina Günther, Frank Edenhofer, Per Hoffmann, Andreas Reif, Sarah Kittel-Schneider
Publié dans: Journal of Clinical Medicine, Numéro 9/12, 2020, Page(s) 4092, ISSN 2077-0383
Éditeur: Multidisciplinary Digital Publishing Institute (MDPI)
DOI: 10.3390/jcm9124092

TARGET-seq: Linking single-cell transcriptomics of human dopaminergic neurons with their target specificity

Auteurs: Alessandro Fiorenzano, Petter Storm, Edoardo Sozzi, Andreas Bruzelius, Sara Corsi, Janko Kajtez, Janitha Mudannayake, Jenny Nelander, Bengt Mattsson, Malin Åkerblom, Tomas Björklund, Anders Björklund, Malin Parmar
Publié dans: Proceedings of the National Academy of Sciences, Numéro 121, 2024, ISSN 0027-8424
Éditeur: National Academy of Sciences
DOI: 10.1073/pnas.2410331121

Stem Cell-Derived Human Striatal Progenitors Innervate Striatal Targets and Alleviate Sensorimotor Deficit in a Rat Model of Huntington Disease

Auteurs: Dario Besusso, Roberta Schellino, Marina Boido, Sara Belloli, Roberta Parolisi, Paola Conforti, Andrea Faedo, Manuel Cernigoj, Ilaria Campus, Angela Laporta, Vittoria Dickinson Bocchi, Valentina Murtaj, Malin Parmar, Paolo Spaiardi, Francesca Talpo, Claudia Maniezzi, Mauro Giuseppe Toselli, Gerardo Biella, Rosa Maria Moresco, Alessandro Vercelli, Annalisa Buffo, Elena Cattaneo
Publié dans: Stem Cell Reports, Numéro 14/5, 2020, Page(s) 876-891, ISSN 2213-6711
Éditeur: Cell Press
DOI: 10.1016/j.stemcr.2020.03.018

Predicting Astrocytic Nuclear Morphology with Machine Learning: A Tree Ensemble Classifier Study

Auteurs: Piercesare Grimaldi; Martina Lorenzati; Marta Ribodino; Elena Signorino; Annalisa Buffo; Paola Berchialla
Publié dans: Applied Sciences, Numéro Volume 13; Numéro 7, 2023, Page(s) 4289, ISSN 2076-3417
Éditeur: MDPI
DOI: 10.3390/app13074289

Visualizing Arc protein dynamics and localization in the mammalian brain using AAV-mediated in situ gene labeling

Auteurs: Martino Avallone, Joaquín Pardo, Tadiwos F Mergiya, Jana Rájová, Atte Räsänen, Marcus Davidsson, Malin Åkerblom, Luis Quintino, Darshan Kumar, Clive R Bramham, Tomas Björklund
Publié dans: Frontiers in Molecular Neuroscience, 2023, ISSN 1662-5099
Éditeur: Frontiers Research Foundation
DOI: 10.3389/fnmol.2023.1140785

A CRISPR-strategy for the generation of a detectable fluorescent hESC reporter line (WAe009-A-37) for the subpallial determinant GSX2

Auteurs: Dario Besusso, Andrea Cossu, Ayat Mohamed, Manuel Cernigoj, Paolo Codega, Maura Galimberti, Ilaria Campus, Paola Conforti, Elena Cattaneo
Publié dans: Stem Cell Research, Numéro 49, 2020, Page(s) 102016, ISSN 1873-5061
Éditeur: Elsevier
DOI: 10.1016/j.scr.2020.102016

Secretome Analyses Identify FKBP4 as a GBA1-Associated Protein in CSF and iPS Cells from Parkinson’s Disease Patients with GBA1 Mutations

Auteurs: Rika Kojima, Wojciech Paslawski, Guochang Lyu, Ernest Arenas, Xiaoqun Zhang, Per Svenningsson
Publié dans: International Journal of Molecular Sciences, Numéro 25, 2024, Page(s) 683, ISSN 1422-0067
Éditeur: Multidisciplinary Digital Publishing Institute (MDPI)
DOI: 10.3390/ijms25010683

In vitro derived medium spiny neurons recapitulate human striatal development and complexity at single-cell resolution.

Auteurs: Paola Conforti, Vittoria Dickinson Bocchi, Ilaria Campus, Linda Scaramuzza, Maura Galimberti, Tiziana Lischetti, Francesca Talpo, Matteo Pedrazzoli, Alessio Murgia, Ivan Ferrari, Chiara Cordiglieri, Alessandra Fasciani, Ernest Arenas, Dan Felsenfeld, Gerardo Biella, Dario Besusso, Elena Cattaneo
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Spatial centrosome proteome of human neural cells uncovers disease-relevant heterogeneity

Auteurs: Adam C. O’Neill, Fatma Uzbas, Giulia Antognolli, Florencia Merino, Kalina Draganova, Alex Jäck, Sirui Zhang, Giorgia Pedini, Julia P. Schessner, Kimberly Cramer, Aloys Schepers, Fabian Metzger, Miriam Esgleas, Pawel Smialowski, Renzo Guerrini, Sven Falk, Regina Feederle, Saskia Freytag, Zefeng Wang, Melanie Bahlo, Ralf Jungmann, Claudia Bagni, Georg H. H. Borner, Stephen P. Robertson, Stefanie
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Bringing Advanced Therapies for Parkinson’s Disease to the Clinic: The Scientist’s Perspective

Auteurs: Mark Tomishima, Agnete Kirkeby
Publié dans: Journal of Parkinsons Disease, 2021, ISSN 1877-7171
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The coding and long noncoding single-cell atlas of the developing human fetal striatum

Auteurs: Vittoria Dickinson Bocchi, Paola Conforti, Elena Vezzoli, Dario Besusso, Claudio Cappadona, Tiziana Lischetti, Maura Galimberti, Valeria Ranzani, Raoul J. P. Bonnal, Marco De Simone, Grazisa Rossetti, Xiaoling He, Kenji Kamimoto, Ira Espuny-Camacho, Andrea Faedo, Federica Gervasoni, Romina Vuono, Samantha A. Morris, Jian Chen, Dan Felsenfeld, Giulio Pavesi, Roger A. Barker, Massimiliano Pagani, El
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The immunogenicity of midbrain dopaminergic neurons and the implications for neural grafting trials in Parkinson's disease.

Auteurs: Shamma Qarin; Sarah Howlett; Joanne L. Jones; Roger A. Barker
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The history and status of dopamine cell therapies for Parkinson's disease

Auteurs: Roger A. Barker, Anders Björklund, Malin Parmar
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Huntington’s disease cellular phenotypes are rescued non-cell autonomously by healthy cells in mosaic telencephalic organoids

Auteurs: Maura Galimberti, Maria R. Nucera, Vittoria D. Bocchi, Paola Conforti, Elena Vezzoli, Matteo Cereda, Camilla Maffezzini, Raffaele Iennaco, Andrea Scolz, Andrea Falqui, Chiara Cordiglieri, Martina Cremona, Ira Espuny-Camacho, Andrea Faedo, Dan P. Felsenfeld, Thomas F. Vogt, Valeria Ranzani, Chiara Zuccato, Dario Besusso, Elena Cattaneo
Publié dans: Nature Communications, Numéro 15, 2025, ISSN 2041-1723
Éditeur: Nature Publishing Group
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<i>SREBP2</i> gene therapy targeting striatal astrocytes ameliorates Huntington’s disease phenotypes

Auteurs: Giulia Birolini, Gianluca Verlengia, Francesca Talpo, Claudia Maniezzi, Lorena Zentilin, Mauro Giacca, Paola Conforti, Chiara Cordiglieri, Claudio Caccia, Valerio Leoni, Franco Taroni, Gerardo Biella, Michele Simonato, Elena Cattaneo, Marta Valenza
Publié dans: Brain, Numéro 144, 2021, Page(s) 3175-3190, ISSN 0006-8950
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Deconvolution of spatial sequencing provides accurate characterization of hESC-derived DA transplants in vivo

Auteurs: Jana Rájová, Marcus Davidsson, Martino Avallone, Morgan Hartnor, Patrick Aldrin-Kirk, Tiago Cardoso, Sara Nolbrant, Annelie Mollbrink, Petter Storm, Andreas Heuer, Malin Parmar, Tomas Björklund
Publié dans: Mol Ther Methods Clin Dev, 2023, ISSN 2329-0501
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Human neural stem cells directly programmed from peripheral blood show functional integration into the adult mouse brain

Auteurs: Lea Jessica Berg, Chung Ku Lee, Hideaki Matsumura, Anke Leinhaas, Rachel Konang, Ali H. Shaib, Pedro Royero, Julia Schlee, Chao Sheng, Heinz Beck, Martin Karl Schwarz, Nils Brose, Jeong Seop Rhee, Oliver Brüstle
Publié dans: Stem Cell Research & Therapy, Numéro 15, 2024, Page(s) 01-06, ISSN 1757-6512
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Brain cholesterol therapy for Huntington's disease – Does it make sense?

Auteurs: Elena Cattaneo, Roger A Barker
Publié dans: Clinical and Translational Medicine, Numéro 14, 2024, ISSN 2001-1326
Éditeur: Wiley Online Library
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Molecular Signature of Astrocytes for Gene Delivery by the Synthetic Adeno-Associated Viral Vector rAAV9P1

Auteurs: Amelie Bauer, Matteo Puglisi, Dennis Nagl, Joel A Schick, Thomas Werner, Andreas Klingl, Jihad El Andari, Veit Hornung, Horst Kessler, Magdalena Götz, Dirk Grimm, Ruth Brack-Werner
Publié dans: Advanced Science, Numéro Volume 9, Numéro 16, 2021, ISSN 2198-3844
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Identifying secreted biomarkers of dopaminergic ventral midbrain progenitor cells

Auteurs: Pedro Rifes, Marc Isaksson, Charlotte Rusimbi, Adrián Ramón Santonja, Jenny Nelander, Thomas Laurell, Agnete Kirkeby
Publié dans: Stem Cell Research and Therapy, 2023, ISSN 1757-6512
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hiPSCs for predictive modelling of neurodegenerative diseases: dreaming the possible.

Auteurs: Pia Rivetti di Val Cervo; Dario Besusso; Paola Conforti; Elena Cattaneo
Publié dans: Nature Reviews. Neurology, Numéro 17, 2021, Page(s) 381–392, ISSN 1759-4758
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hESC-derived striatal progenitors grafted into a Huntington's disease rat model support long-term functional motor recovery by differentiating, self-organizing and connecting into the lesioned striatum

Auteurs: Schellino R, Besusso D, Parolisi R, Gómez-González GB, Dallere S, Scaramuzza L, Ribodino M, Campus I, Conforti P, Parmar M, Boido M, Cattaneo E, Buffo A
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Direct neuronal reprogramming of mouse astrocytes is associated with multiscale epigenome remodeling and requires Yy1

Auteurs: Allwyn Pereira, Jeisimhan Diwakar, Giacomo Masserdotti, Sude Beşkardeş, Tatiana Simon, Younju So, Lucía Martín-Loarte, Franziska Bergemann, Lakshmy Vasan, Tamas Schauer, Anna Danese, Riccardo Bocchi, Maria Colomé-Tatché, Carol Schuurmans, Anna Philpott, Tobias Straub, Boyan Bonev, Magdalena Götz
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Molecular diversity of diencephalic astrocytes reveals adult astrogenesis regulated by Smad4

Auteurs: Stefanie Ohlig, Solène Clavreul, Manja Thorwirth, Tatiana Simon-Ebert, Riccardo Bocchi, Sabine Ulbricht, Nirmal Kannayian, Moritz Rossner, Swetlana Sirko, Pawel Smialowski, Judith Fischer-Sternjak , Magdalena Götz
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In vitro modeling of the human dopaminergic system using spatially arranged ventral midbrain–striatum–cortex assembloids

Auteurs: Daniel Reumann, Christian Krauditsch, Maria Novatchkova, Edoardo Sozzi, Sakurako Nagumo Wong, Michael Zabolocki, Marthe Priouret, Balint Doleschall, Kaja I. Ritzau-Reid, Marielle Piber, Ilaria Morassut, Charles Fieseler, Alessandro Fiorenzano, Molly M. Stevens, Manuel Zimmer, Cedric Bardy, Malin Parmar & Jürgen A. Knoblich
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Éditeur: Nature Publishing Group
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Stem Cell Revolutions: An alliance for Parkinson’s disease driven by European Commission consortia

Auteurs: Elena Cattaneo, Dario Besusso
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Method to combat Parkinson’s disease by astrocyte-to-neuron conversion

Auteurs: Ernest Arenas
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Multimodal synapse analysis reveals restricted integration of transplanted neurons remodeled by TREM2

Auteurs: Yvette Zarb, Manja Thorwirth, Oskar Markkula, Emanuele Paoli, Maria Fernanda Martinez-Reza, Mihail Todorov, Christina Koupoutidou, Veronika Schwarz, Georg Kislinger, Alexandra Mezydlo, Matteo Puglisi, Michael Willem, Jovica Ninkovic, Karl-Klaus Conzelmann, Martina Schifferer, Christian Haass, Martin Kerschensteiner, Ruben Portugues, Conny Kopp-Scheinpflug, Magdalena Goetz
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APCDD1 outperforms other dopaminergic cell surface markers in benchmarking study

Auteurs: Agnete Kirkeby, Alrik Schörling, Erno Hänninen, Pedro Rifes, Alison Salvador, Amalie Holm, Gaurav Singh Rathore, Jenny Nelander, Josefine Christiansen, Simone Møller Jensen, Jonathan Niclis, Emma Gustafsson, Patrick Aldrin-Kirk, Yu Zhang, Malin Parmar
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Generation of human appetite-regulating neurons and tanycytes from stem cells

Auteurs: Zehra Abay-Nørgaard, Anika K Müller, Erno Hänninen, Dylan Rausch, Louise Piilgaard, Jens Bager Christensen, Sofie Peeters, Alrik L. Schörling, Alison Salvador, Viktoriia Nikulina, Yuan Li, Janko Kajtez, Tune H Pers, Agnete Kirkeby
Publié dans: BioRxiv, 2024
Éditeur: Cold Spring Harbor Laboratory
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The immunological profile of RC17 hESC-derived dopaminergic neural progenitor cells in vitro: implications for the STEM-PD clinical trial

Auteurs: Annabel J Curle, Shaline V Fazal, Shamma Qarin, Sarah K Howlett, Xiaoling He, Roger A Barker, Joanne L Jones
Publié dans: biorxiv, 2024, ISSN 2692-8205
Éditeur: Cold Spring Harbor Laboratory
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Patterning effects of FGF17 and cAMP on generation of dopaminergic progenitors for cell replacement therapy in Parkinson’s disease

Auteurs: Amalie Holm Nygaard, Alrik L. Schörling, Zehra Abay-Nørgaard, Erno Hänninen, Yuan Li, Adrian Santoja, Gaurav Singh Rathore, Alison Salvador, Charlotte Rusimbi, Yu Zhang, Agnete Kirkeby
Publié dans: BioRxiv, 2024
Éditeur: Cold Spring Harbor Laboratory
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TGIF2 is a major regulator of neural stem cell fate and neurogenic priming

Auteurs: Yiling Li, Anthi C. Krontira, Franziska Vierl, Maria L. Richter, Weixu Wang, Juliane Merl-Pham, Fabian J. Theis, Stefanie M. Hauck, Magdalena Götz
Publié dans: BioRxiv, 2025
Éditeur: Cold Spring Harbor Laboratory
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Generation of human cerebral organoids with a structured outer subventricular zone

Auteurs: Ryan Walsh; Elisa Giacomelli; Gabriele Ciceri; Chelsea Rittenhouse; Maura Galimberti; Youjun Wu; James Muller; Elena Vezzoli; Johannes Jungverdorben; Ting Zhou; Roger A Barker; Elena Cattaneo; Lorenz Studer; Arianna Baggiolini
Publié dans: bioRxiv, 2023, ISSN 2692-8205
Éditeur: Cold Spring Harbor Laboratory
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Scaling of cell and gene therapies to population

Auteurs: Sebastian Knöbel, Andreas Bosio
Publié dans: Handbook of Clinical Neurology, Cell and Gene Therapies for Neurologic Diseases, 2024, Page(s) 145-154
Éditeur: Elsevier
DOI: 10.1016/b978-0-323-90120-8.00012-5

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