Novel Strategies for Cell-based Neural Reconstruction

CORDIS provides links to public deliverables and publications of HORIZON projects.

Links to deliverables and publications from FP7 projects, as well as links to some specific result types such as dataset and software, are dynamically retrieved from OpenAIRE .

Deliverables

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

Author(s): Giacomoni J, Habekost M, Cepeda-Prado E, Mattsson B, Ottosson DR, Parmar M, Kajtez J
Published in: STAR Protoc, Issue Mar 17;4(1):102041, 2023, ISSN 2666-1667
Publisher: Cell Press
DOI: 10.1016/j.xpro.2022.102041

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

Author(s): Nishimura K, Ásgrímsdóttir ES, Yang S, Arenas E
Published in: STAR Protocols, Issue Volume 4, Issue 3, 15 September 2023, 2023, ISSN 2666-1667
Publisher: Cell Press Elsevier Inc
DOI: 10.1016/j.xpro.2023.102355

Spatial Centrosome Proteomic Profiling of Human iPSC-derived Neural Cells

Author(s): Fatma Uzbas, Adam O’Neill
Published in: BIO-PROTOCOL, Issue 13, 2024, ISSN 2331-8325
Publisher: Bio-protocol LLC
DOI: 10.21769/bioprotoc.4812

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

Author(s): Marco Fogli, Giulia Nato, Philip Greulich, Jacopo Pinto, Marta Ribodino, Gregorio Valsania, Paolo Peretto, Annalisa Buffo, Federico Luzzati
Published in: Stem Cell Reports, Issue 19, 2024, Page(s) 1432-1450, ISSN 2213-6711
Publisher: 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

Author(s): 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
Published in: Stem Cell Reports, Issue Vol. 18 Issue 1, 2023, Page(s) 337-353, ISSN 2213-6711
Publisher: 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

Author(s): 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
Published in: Cell, Issue 186(26), 2023, Page(s) 5766-5783.e25, ISSN 1097-4172
Publisher: Cell Press
DOI: 10.1016/j.cell.2023.11.028

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

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

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

Author(s): 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
Published in: Mol Syst Biol., 2022, Page(s) e11129, ISSN 1744-4292
Publisher: Nature Publishing Group
DOI: 10.15252/msb.202211129

Generation of human cerebral organoids with a structured outer subventricular zone

Author(s): 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
Published in: Cell Reports, Issue 43(4), 2024, Page(s) 114031, ISSN 2211-1247
Publisher: Cell Press
DOI: 10.1016/j.celrep.2024.114031

Stem cell programming – prospects for perinatal medicine

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

Generation of Human Ventral Midbrain Organoids Derived from Pluripotent Stem Cells

Author(s): Edoardo Sozzi, Fredrik Nilsson, Janko Kajtez, Malin Parmar, Alessandro Fiorenzano
Published in: Current protocols., 2022, ISSN 2691-1299
Publisher: 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

Author(s): 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
Published in: Cell Reports, Issue 36/3, 2021, Page(s) 109409, ISSN 2211-1247
Publisher: Cell Press
DOI: 10.1016/j.celrep.2021.109409

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

Author(s): 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
Published in: Nature Medicine, Issue 29, 2023, Page(s) 149–3161, ISSN 1546-170X
Publisher: 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

Author(s): Aldrin-Kirk P, Åkerblom M, Cardoso T, Nolbrant S, Adler AF, Liu X, Heuer A, Davidsson M, Parmar M, Björklund T
Published in: Stem Cell Reports, 2022, ISSN 2213-6711
Publisher: 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

Author(s): 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
Published in: Stem Cells, 2025, ISSN 1066-5099
Publisher: AlphaMed Press Inc
DOI: 10.1093/stmcls/sxaf004

Transcriptomic diversity of cell types across the adult human brain

Author(s): 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
Published in: Science, Issue Vol 382, Issue 6667, 13 October 2023, 2023, ISSN 0036-8075
Publisher: 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

Author(s): Maria Cruz-Santos, Lucia Fernandez Cardo, Meng Li
Published in: Cells, Issue 11(5), 853, 2022, ISSN 2073-4409
Publisher: MDPI
DOI: 10.3390/cells11050853

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

Author(s): 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
Published in: Frontiers in cell and developmental biology, 2022, ISSN 2296-634X
Publisher: 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

Author(s): Jianbin Wen, Michael Peitz, Oliver Brüstle
Published in: Journal of Neuroscience Methods, Issue 373, 2022, ISSN 0165-0270
Publisher: Elsevier BV
DOI: 10.1016/j.jneumeth.2022.109562

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

Author(s): Emilía Sif Ásgrímsdóttir, Ernest Arenas
Published in: Frontiers in Cell and Developmental Biology, Issue 8, 2020, ISSN 2296-634X
Publisher: 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?

Author(s): Malin Parmar, Anders Björklund, Tomas Björklund
Published in: Current Opinion in Genetics & Development, 2021, Page(s) 76-82, ISSN 0959-437X
Publisher: 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

Author(s): 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
Published in: Glia, 2024, ISSN 0894-1491
Publisher: John Wiley & Sons Inc.
DOI: 10.1002/glia.24645

Brain injury environment critically influences the connectivity of transplanted neurons

Author(s): Sofia Grade, Judith Thomas, Yvette Zarb, Manja Thorwirth, Karl-Klaus Conzelmann, Stefanie M. Hauck, Magdalena Götz
Published in: Science Advances, Issue 8, 2024, ISSN 2375-2548
Publisher: AAAS
DOI: 10.1126/sciadv.abg9445

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

Author(s): Juan Eduardo Rodriguez-Gatica; Vira Iefremova; Liubov Sokhranyaeva; Si Wah Christina Au Yeung; Yannik Breitkreuz; Oliver Brüstle; Martin Karl Schwarz; Ulrich Kubitscheck
Published in: Development, Issue 149, 2022, ISSN 0950-1991
Publisher: The Company of Biologists Ltd.
DOI: 10.1242/dev.200439

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

Author(s): Riccardo Bocchi, Giacomo Masserdotti, Magdalena Götz
Published in: Neuron, Issue 110(3), 2022, Page(s) 366-393, ISSN 0896-6273
Publisher: 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

Author(s): 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
Published in: American Journal of Medical Genetics Part B: Neuropsychiatric Genetics, Issue 189(7-8), 2022, ISSN 1552-4876
Publisher: 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

Author(s): Maria Cruz Santos, Meng Li
Published in: Neuronal Signaling, Issue 5 (4), 2021, ISSN 2059-6553
Publisher: Portland Press
DOI: 10.1042/ns20210020

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

Author(s): Josefine Rågård Christiansen, Agnete Kirkeby
Published in: Development, Issue 151, 2024, ISSN 0950-1991
Publisher: The Company of Biologists Ltd.
DOI: 10.1242/dev.202067

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

Author(s): Ben Vermaercke, Ryohei Iwata, Keimpe Wierda, Leïla Boubakar, Paula Rodriguez, Martyna Ditkowska, Vincent Bonin, Pierre Vanderhaeghen
Published in: Neuron, Issue 112, 2024, Page(s) 3058-3068.e8, ISSN 0896-6273
Publisher: 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

Author(s): 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
Published in: Nat Commun, 2021, ISSN 2041-1723
Publisher: 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

Author(s): Emilía Sif Ásgrímsdóttir, Ernest Arenas
Published in: Stem Cell Research, Issue 81, 2025, Page(s) 103523, ISSN 1873-5061
Publisher: 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

Author(s): 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
Published in: Neuron, Issue 112, 2024, Page(s) 1117-1132.e9, ISSN 0896-6273
Publisher: Cell Press
DOI: 10.1016/j.neuron.2023.12.020

Comprehensive cell atlas of the first-trimester developing human brain

Author(s): 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
Published in: Science, Issue Vol 382, Issue 6667, 13 October 2023, 2023, ISSN 0036-8075
Publisher: 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

Author(s): Maqsood Ahmed, Matthew J. Owens, Enrique M. Toledo, Ernest Arenas, Mark Bradley, Charles ffrench-Constant
Published in: Frontiers in Cell and Developmental Biology, Issue Volume 9 - 2021, 2021, ISSN 2296-634X
Publisher: 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

Author(s): Matteo Puglisi, Chu Lan Lao, Gulzar Wani, Giacomo Masserdotti, Riccardo Bocchi, Magdalena Götz
Published in: Cells, Issue 13, 2024, Page(s) 1408, ISSN 2073-4409
Publisher: MDPI
DOI: 10.3390/cells13171408

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

Author(s): 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
Published in: Neuron, Issue 112, 2024, Page(s) 3602-3617.e9, ISSN 0896-6273
Publisher: 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

Author(s): Petter Storm, Yu Zhang, Fredrik Nilsson, Alessandro Fiorenzano, Niklas Krausse, Malin Åkerblom, Marcus Davidsson, Joan Yuan, Agnete Kirkeby, Tomas Björklund, Malin Parmar
Published in: Science Advances, Issue 10, 2024, ISSN 2375-2548
Publisher: AAAS
DOI: 10.1126/sciadv.adn3057

Astrocyte heterogeneity reveals region-specific astrogenesis in the white matter

Author(s): 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
Published in: Nature Neuroscience, 2025, ISSN 1097-6256
Publisher: 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

Author(s): 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
Published in: Cell Stem Cell, 2023, ISSN 1934-5909
Publisher: 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

Author(s): Pedro Rifes, Janko Kajtez, Josefine Rågård Christiansen, Alrik Schörling, Gaurav Singh Rathore, Daniel A. Wolf, Andreas Heuer, Agnete Kirkeby
Published in: Stem Cell Reports, Issue Volume 19, Issue 6, 2024, Page(s) 830-838, ISSN 2213-6711
Publisher: 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

Author(s): 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
Published in: Scientific Report, Issue 12(1), 2022, ISSN 2045-2322
Publisher: Nature Publishing Group
DOI: 10.1038/s41598-022-12404-0

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

Author(s): 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
Published in: Journal of Clinical Medicine, Issue 9/12, 2020, Page(s) 4092, ISSN 2077-0383
Publisher: Multidisciplinary Digital Publishing Institute (MDPI)
DOI: 10.3390/jcm9124092

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

Author(s): 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
Published in: Proceedings of the National Academy of Sciences, Issue 121, 2024, ISSN 0027-8424
Publisher: 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

Author(s): 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
Published in: Stem Cell Reports, Issue 14/5, 2020, Page(s) 876-891, ISSN 2213-6711
Publisher: Cell Press
DOI: 10.1016/j.stemcr.2020.03.018

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

Author(s): Piercesare Grimaldi; Martina Lorenzati; Marta Ribodino; Elena Signorino; Annalisa Buffo; Paola Berchialla
Published in: Applied Sciences, Issue Volume 13; Issue 7, 2023, Page(s) 4289, ISSN 2076-3417
Publisher: MDPI
DOI: 10.3390/app13074289

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

Author(s): Martino Avallone, Joaquín Pardo, Tadiwos F Mergiya, Jana Rájová, Atte Räsänen, Marcus Davidsson, Malin Åkerblom, Luis Quintino, Darshan Kumar, Clive R Bramham, Tomas Björklund
Published in: Frontiers in Molecular Neuroscience, 2023, ISSN 1662-5099
Publisher: 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

Author(s): Dario Besusso, Andrea Cossu, Ayat Mohamed, Manuel Cernigoj, Paolo Codega, Maura Galimberti, Ilaria Campus, Paola Conforti, Elena Cattaneo
Published in: Stem Cell Research, Issue 49, 2020, Page(s) 102016, ISSN 1873-5061
Publisher: 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

Author(s): Rika Kojima, Wojciech Paslawski, Guochang Lyu, Ernest Arenas, Xiaoqun Zhang, Per Svenningsson
Published in: International Journal of Molecular Sciences, Issue 25, 2024, Page(s) 683, ISSN 1422-0067
Publisher: 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.

Author(s): 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
Published in: Cell Reports Methods, Issue Volume 2, Issue 12, 2022, ISSN 2667-2375
Publisher: Cell Press
DOI: 10.1016/j.crmeth.2022.100367

Spatial centrosome proteome of human neural cells uncovers disease-relevant heterogeneity

Author(s): 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
Published in: Science, Issue 376, 2024, ISSN 0036-8075
Publisher: American Association for the Advancement of Science
DOI: 10.1126/science.abf9088

Bringing Advanced Therapies for Parkinson’s Disease to the Clinic: The Scientist’s Perspective

Author(s): Mark Tomishima, Agnete Kirkeby
Published in: Journal of Parkinsons Disease, 2021, ISSN 1877-7171
Publisher: IOS Press
DOI: 10.3233/jpd-212685

The coding and long noncoding single-cell atlas of the developing human fetal striatum

Author(s): 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
Published in: Science, Issue 372/6542, 2021, Page(s) eabf5759, ISSN 0036-8075
Publisher: American Association for the Advancement of Science
DOI: 10.1126/science.abf5759

The immunogenicity of midbrain dopaminergic neurons and the implications for neural grafting trials in Parkinson's disease.

Author(s): Shamma Qarin; Sarah Howlett; Joanne L. Jones; Roger A. Barker
Published in: Neuronal Signaling, Issue 5 (3), 2021, ISSN 2059-6553
Publisher: Portland Press
DOI: 10.1042/ns20200083

The history and status of dopamine cell therapies for Parkinson's disease

Author(s): Roger A. Barker, Anders Björklund, Malin Parmar
Published in: BioEssays, Issue 46, 2024, ISSN 0265-9247
Publisher: John Wiley & Sons Inc.
DOI: 10.1002/bies.202400118

Huntington’s disease cellular phenotypes are rescued non-cell autonomously by healthy cells in mosaic telencephalic organoids

Author(s): 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
Published in: Nature Communications, Issue 15, 2025, ISSN 2041-1723
Publisher: Nature Publishing Group
DOI: 10.1038/s41467-024-50877-x

<i>SREBP2</i> gene therapy targeting striatal astrocytes ameliorates Huntington’s disease phenotypes

Author(s): 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
Published in: Brain, Issue 144, 2021, Page(s) 3175-3190, ISSN 0006-8950
Publisher: Oxford University Press
DOI: 10.1093/brain/awab186

Deconvolution of spatial sequencing provides accurate characterization of hESC-derived DA transplants in vivo

Author(s): 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
Published in: Mol Ther Methods Clin Dev, 2023, ISSN 2329-0501
Publisher: Nature Publishing Group
DOI: 10.1016/j.omtm.2023.04.008

Human neural stem cells directly programmed from peripheral blood show functional integration into the adult mouse brain

Author(s): 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
Published in: Stem Cell Research & Therapy, Issue 15, 2024, Page(s) 01-06, ISSN 1757-6512
Publisher: BioMed Central
DOI: 10.1186/s13287-024-04110-7

Brain cholesterol therapy for Huntington's disease – Does it make sense?

Author(s): Elena Cattaneo, Roger A Barker
Published in: Clinical and Translational Medicine, Issue 14, 2024, ISSN 2001-1326
Publisher: Wiley Online Library
DOI: 10.1002/ctm2.1746

Molecular Signature of Astrocytes for Gene Delivery by the Synthetic Adeno-Associated Viral Vector rAAV9P1

Author(s): 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
Published in: Advanced Science, Issue Volume 9, Issue 16, 2021, ISSN 2198-3844
Publisher: Wiley-VCH
DOI: 10.1002/advs.202104979

Identifying secreted biomarkers of dopaminergic ventral midbrain progenitor cells

Author(s): Pedro Rifes, Marc Isaksson, Charlotte Rusimbi, Adrián Ramón Santonja, Jenny Nelander, Thomas Laurell, Agnete Kirkeby
Published in: Stem Cell Research and Therapy, 2023, ISSN 1757-6512
Publisher: BioMed Central
DOI: 10.1186/s13287-023-03580-5

hiPSCs for predictive modelling of neurodegenerative diseases: dreaming the possible.

Author(s): Pia Rivetti di Val Cervo; Dario Besusso; Paola Conforti; Elena Cattaneo
Published in: Nature Reviews. Neurology, Issue 17, 2021, Page(s) 381–392, ISSN 1759-4758
Publisher: Nature Publishing Group
DOI: 10.1038/s41582-021-00465-0

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

Author(s): 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
Published in: Stem Cell Res Ther, Issue Jul 28;14(1), 2023, Page(s) Article number: 189, ISSN 1757-6512
Publisher: BioMed Central
DOI: 10.1186/s13287-023-03422-4

Direct neuronal reprogramming of mouse astrocytes is associated with multiscale epigenome remodeling and requires Yy1

Author(s): 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
Published in: Nature Neuroscience, Issue 27, 2024, Page(s) 1260-1273, ISSN 1097-6256
Publisher: Nature Publishing Group
DOI: 10.1038/s41593-024-01677-5

Molecular diversity of diencephalic astrocytes reveals adult astrogenesis regulated by Smad4

Author(s): 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
Published in: EMBO J, Issue 40(21), 2021, Page(s) e107532, ISSN 1460-2075
Publisher: EMBO Press
DOI: 10.15252/embj.2020107532

In vitro modeling of the human dopaminergic system using spatially arranged ventral midbrain–striatum–cortex assembloids

Author(s): 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
Published in: Nature Methods, 2023, ISSN 1548-7091
Publisher: Nature Publishing Group
DOI: 10.1038/s41592-023-02080-x

Stem Cell Revolutions: An alliance for Parkinson’s disease driven by European Commission consortia

Author(s): Elena Cattaneo, Dario Besusso
Published in: Pharmacological Research, Issue 212, 2025, Page(s) 107569, ISSN 1043-6618
Publisher: Academic Press
DOI: 10.1016/j.phrs.2024.107569

Method to combat Parkinson’s disease by astrocyte-to-neuron conversion

Author(s): Ernest Arenas
Published in: Nature, Issue 582/7813, 2020, Page(s) 489-490, ISSN 0028-0836
Publisher: Nature Publishing Group
DOI: 10.1038/d41586-020-01817-4

Multimodal synapse analysis reveals restricted integration of transplanted neurons remodeled by TREM2

Author(s): 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
Published in: BioRxiv, 2025
Publisher: Cold Spring Harbor Laboratory
DOI: 10.1101/2025.01.31.635250

APCDD1 outperforms other dopaminergic cell surface markers in benchmarking study

Author(s): 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
Published in: Research Square, 2023, ISSN 2693-5015
Publisher: Research Square Platform LLC
DOI: 10.21203/rs.3.rs-3266974/v1

Generation of human appetite-regulating neurons and tanycytes from stem cells

Author(s): 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
Published in: BioRxiv, 2024
Publisher: Cold Spring Harbor Laboratory
DOI: 10.1101/2024.07.11.603039

The immunological profile of RC17 hESC-derived dopaminergic neural progenitor cells in vitro: implications for the STEM-PD clinical trial

Author(s): Annabel J Curle, Shaline V Fazal, Shamma Qarin, Sarah K Howlett, Xiaoling He, Roger A Barker, Joanne L Jones
Published in: biorxiv, 2024, ISSN 2692-8205
Publisher: Cold Spring Harbor Laboratory
DOI: 10.1101/2024.01.23.576826

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

Author(s): 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
Published in: BioRxiv, 2024
Publisher: Cold Spring Harbor Laboratory
DOI: 10.1101/2024.05.08.593131

TGIF2 is a major regulator of neural stem cell fate and neurogenic priming

Author(s): Yiling Li, Anthi C. Krontira, Franziska Vierl, Maria L. Richter, Weixu Wang, Juliane Merl-Pham, Fabian J. Theis, Stefanie M. Hauck, Magdalena Götz
Published in: BioRxiv, 2025
Publisher: Cold Spring Harbor Laboratory
DOI: 10.1101/2025.02.13.635953

Generation of human cerebral organoids with a structured outer subventricular zone

Author(s): 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
Published in: bioRxiv, 2023, ISSN 2692-8205
Publisher: Cold Spring Harbor Laboratory
DOI: 10.1101/2023.02.17.528906

Scaling of cell and gene therapies to population

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

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