Novel Strategies for Cell-based Neural Reconstruction

CORDIS proporciona enlaces a los documentos públicos y las publicaciones de los proyectos de los programas marco HORIZONTE.

Los enlaces a los documentos y las publicaciones de los proyectos del Séptimo Programa Marco, así como los enlaces a algunos tipos de resultados específicos, como conjuntos de datos y «software», se obtienen dinámicamente de OpenAIRE .

Resultado final

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

Publicaciones

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

Autores: Giacomoni J, Habekost M, Cepeda-Prado E, Mattsson B, Ottosson DR, Parmar M, Kajtez J
Publicado en: STAR Protoc, Edición Mar 17;4(1):102041, 2023, ISSN 2666-1667
Editor: Cell Press
DOI: 10.1016/j.xpro.2022.102041

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

Autores: Nishimura K, Ásgrímsdóttir ES, Yang S, Arenas E
Publicado en: STAR Protocols, Edición Volume 4, Edición 3, 15 September 2023, 2023, ISSN 2666-1667
Editor: Cell Press Elsevier Inc
DOI: 10.1016/j.xpro.2023.102355

Spatial Centrosome Proteomic Profiling of Human iPSC-derived Neural Cells

Autores: Fatma Uzbas, Adam O’Neill
Publicado en: BIO-PROTOCOL, Edición 13, 2024, ISSN 2331-8325
Editor: Bio-protocol LLC
DOI: 10.21769/bioprotoc.4812

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

Autores: Marco Fogli, Giulia Nato, Philip Greulich, Jacopo Pinto, Marta Ribodino, Gregorio Valsania, Paolo Peretto, Annalisa Buffo, Federico Luzzati
Publicado en: Stem Cell Reports, Edición 19, 2024, Página(s) 1432-1450, ISSN 2213-6711
Editor: 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

Autores: 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
Publicado en: Stem Cell Reports, Edición Vol. 18 Edición 1, 2023, Página(s) 337-353, ISSN 2213-6711
Editor: 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

Autores: 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
Publicado en: Cell, Edición 186(26), 2023, Página(s) 5766-5783.e25, ISSN 1097-4172
Editor: Cell Press
DOI: 10.1016/j.cell.2023.11.028

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

Autores: Thomas J, Martinez-Reza MF, Thorwirth M, Zarb Y, Conzelmann KK, Hauck SM, Grade S, Götz M
Publicado en: Science Advances, Edición 10;8(23), 2022, Página(s) eabg9287, ISSN 2375-2548
Editor: American Association for the Advancement of Science
DOI: 10.1126/sciadv.abg9287

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

Autores: 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
Publicado en: Mol Syst Biol., 2022, Página(s) e11129, ISSN 1744-4292
Editor: Nature Publishing Group
DOI: 10.15252/msb.202211129

Generation of human cerebral organoids with a structured outer subventricular zone

Autores: 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
Publicado en: Cell Reports, Edición 43(4), 2024, Página(s) 114031, ISSN 2211-1247
Editor: Cell Press
DOI: 10.1016/j.celrep.2024.114031

Stem cell programming – prospects for perinatal medicine

Autores: Berg, L.J., Brüstle, O
Publicado en: Journal of Perinatal Medicine, Edición 2023 Feb 22;51(6), 2023, Página(s) 730-736, ISSN 0300-5577
Editor: Walter de Gruyter GmbH & Co. KG
DOI: 10.1515/jpm-2022-0575

Generation of Human Ventral Midbrain Organoids Derived from Pluripotent Stem Cells

Autores: Edoardo Sozzi, Fredrik Nilsson, Janko Kajtez, Malin Parmar, Alessandro Fiorenzano
Publicado en: Current protocols., 2022, ISSN 2691-1299
Editor: 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

Autores: 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
Publicado en: Cell Reports, Edición 36/3, 2021, Página(s) 109409, ISSN 2211-1247
Editor: Cell Press
DOI: 10.1016/j.celrep.2021.109409

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

Autores: 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
Publicado en: Nature Medicine, Edición 29, 2023, Página(s) 149–3161, ISSN 1546-170X
Editor: 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

Autores: Aldrin-Kirk P, Åkerblom M, Cardoso T, Nolbrant S, Adler AF, Liu X, Heuer A, Davidsson M, Parmar M, Björklund T
Publicado en: Stem Cell Reports, 2022, ISSN 2213-6711
Editor: 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

Autores: 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
Publicado en: Stem Cells, 2025, ISSN 1066-5099
Editor: AlphaMed Press Inc
DOI: 10.1093/stmcls/sxaf004

Transcriptomic diversity of cell types across the adult human brain

Autores: 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
Publicado en: Science, Edición Vol 382, Edición 6667, 13 October 2023, 2023, ISSN 0036-8075
Editor: 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

Autores: Maria Cruz-Santos, Lucia Fernandez Cardo, Meng Li
Publicado en: Cells, Edición 11(5), 853, 2022, ISSN 2073-4409
Editor: MDPI
DOI: 10.3390/cells11050853

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

Autores: 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
Publicado en: Frontiers in cell and developmental biology, 2022, ISSN 2296-634X
Editor: 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

Autores: Jianbin Wen, Michael Peitz, Oliver Brüstle
Publicado en: Journal of Neuroscience Methods, Edición 373, 2022, ISSN 0165-0270
Editor: Elsevier BV
DOI: 10.1016/j.jneumeth.2022.109562

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

Autores: Emilía Sif Ásgrímsdóttir, Ernest Arenas
Publicado en: Frontiers in Cell and Developmental Biology, Edición 8, 2020, ISSN 2296-634X
Editor: 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?

Autores: Malin Parmar, Anders Björklund, Tomas Björklund
Publicado en: Current Opinion in Genetics & Development, 2021, Página(s) 76-82, ISSN 0959-437X
Editor: 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

Autores: 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
Publicado en: Glia, 2024, ISSN 0894-1491
Editor: John Wiley & Sons Inc.
DOI: 10.1002/glia.24645

Brain injury environment critically influences the connectivity of transplanted neurons

Autores: Sofia Grade, Judith Thomas, Yvette Zarb, Manja Thorwirth, Karl-Klaus Conzelmann, Stefanie M. Hauck, Magdalena Götz
Publicado en: Science Advances, Edición 8, 2024, ISSN 2375-2548
Editor: AAAS
DOI: 10.1126/sciadv.abg9445

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

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

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

Autores: Riccardo Bocchi, Giacomo Masserdotti, Magdalena Götz
Publicado en: Neuron, Edición 110(3), 2022, Página(s) 366-393, ISSN 0896-6273
Editor: 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

Autores: 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
Publicado en: American Journal of Medical Genetics Part B: Neuropsychiatric Genetics, Edición 189(7-8), 2022, ISSN 1552-4876
Editor: 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

Autores: Maria Cruz Santos, Meng Li
Publicado en: Neuronal Signaling, Edición 5 (4), 2021, ISSN 2059-6553
Editor: Portland Press
DOI: 10.1042/ns20210020

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

Autores: Josefine Rågård Christiansen, Agnete Kirkeby
Publicado en: Development, Edición 151, 2024, ISSN 0950-1991
Editor: The Company of Biologists Ltd.
DOI: 10.1242/dev.202067

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

Autores: Ben Vermaercke, Ryohei Iwata, Keimpe Wierda, Leïla Boubakar, Paula Rodriguez, Martyna Ditkowska, Vincent Bonin, Pierre Vanderhaeghen
Publicado en: Neuron, Edición 112, 2024, Página(s) 3058-3068.e8, ISSN 0896-6273
Editor: 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

Autores: 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
Publicado en: Nat Commun, 2021, ISSN 2041-1723
Editor: 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

Autores: Emilía Sif Ásgrímsdóttir, Ernest Arenas
Publicado en: Stem Cell Research, Edición 81, 2025, Página(s) 103523, ISSN 1873-5061
Editor: 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

Autores: 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
Publicado en: Neuron, Edición 112, 2024, Página(s) 1117-1132.e9, ISSN 0896-6273
Editor: Cell Press
DOI: 10.1016/j.neuron.2023.12.020

Comprehensive cell atlas of the first-trimester developing human brain

Autores: 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
Publicado en: Science, Edición Vol 382, Edición 6667, 13 October 2023, 2023, ISSN 0036-8075
Editor: 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

Autores: Maqsood Ahmed, Matthew J. Owens, Enrique M. Toledo, Ernest Arenas, Mark Bradley, Charles ffrench-Constant
Publicado en: Frontiers in Cell and Developmental Biology, Edición Volume 9 - 2021, 2021, ISSN 2296-634X
Editor: 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

Autores: Matteo Puglisi, Chu Lan Lao, Gulzar Wani, Giacomo Masserdotti, Riccardo Bocchi, Magdalena Götz
Publicado en: Cells, Edición 13, 2024, Página(s) 1408, ISSN 2073-4409
Editor: MDPI
DOI: 10.3390/cells13171408

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

Autores: 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
Publicado en: Neuron, Edición 112, 2024, Página(s) 3602-3617.e9, ISSN 0896-6273
Editor: 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

Autores: Petter Storm, Yu Zhang, Fredrik Nilsson, Alessandro Fiorenzano, Niklas Krausse, Malin Åkerblom, Marcus Davidsson, Joan Yuan, Agnete Kirkeby, Tomas Björklund, Malin Parmar
Publicado en: Science Advances, Edición 10, 2024, ISSN 2375-2548
Editor: AAAS
DOI: 10.1126/sciadv.adn3057

Astrocyte heterogeneity reveals region-specific astrogenesis in the white matter

Autores: 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
Publicado en: Nature Neuroscience, 2025, ISSN 1097-6256
Editor: 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

Autores: 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
Publicado en: Cell Stem Cell, 2023, ISSN 1934-5909
Editor: 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

Autores: Pedro Rifes, Janko Kajtez, Josefine Rågård Christiansen, Alrik Schörling, Gaurav Singh Rathore, Daniel A. Wolf, Andreas Heuer, Agnete Kirkeby
Publicado en: Stem Cell Reports, Edición Volume 19, Edición 6, 2024, Página(s) 830-838, ISSN 2213-6711
Editor: 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

Autores: 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
Publicado en: Scientific Report, Edición 12(1), 2022, ISSN 2045-2322
Editor: Nature Publishing Group
DOI: 10.1038/s41598-022-12404-0

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

Autores: 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
Publicado en: Journal of Clinical Medicine, Edición 9/12, 2020, Página(s) 4092, ISSN 2077-0383
Editor: Multidisciplinary Digital Publishing Institute (MDPI)
DOI: 10.3390/jcm9124092

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

Autores: 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
Publicado en: Proceedings of the National Academy of Sciences, Edición 121, 2024, ISSN 0027-8424
Editor: 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

Autores: 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
Publicado en: Stem Cell Reports, Edición 14/5, 2020, Página(s) 876-891, ISSN 2213-6711
Editor: Cell Press
DOI: 10.1016/j.stemcr.2020.03.018

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

Autores: Piercesare Grimaldi; Martina Lorenzati; Marta Ribodino; Elena Signorino; Annalisa Buffo; Paola Berchialla
Publicado en: Applied Sciences, Edición Volume 13; Edición 7, 2023, Página(s) 4289, ISSN 2076-3417
Editor: MDPI
DOI: 10.3390/app13074289

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

Autores: Martino Avallone, Joaquín Pardo, Tadiwos F Mergiya, Jana Rájová, Atte Räsänen, Marcus Davidsson, Malin Åkerblom, Luis Quintino, Darshan Kumar, Clive R Bramham, Tomas Björklund
Publicado en: Frontiers in Molecular Neuroscience, 2023, ISSN 1662-5099
Editor: 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

Autores: Dario Besusso, Andrea Cossu, Ayat Mohamed, Manuel Cernigoj, Paolo Codega, Maura Galimberti, Ilaria Campus, Paola Conforti, Elena Cattaneo
Publicado en: Stem Cell Research, Edición 49, 2020, Página(s) 102016, ISSN 1873-5061
Editor: 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

Autores: Rika Kojima, Wojciech Paslawski, Guochang Lyu, Ernest Arenas, Xiaoqun Zhang, Per Svenningsson
Publicado en: International Journal of Molecular Sciences, Edición 25, 2024, Página(s) 683, ISSN 1422-0067
Editor: 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.

Autores: 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
Publicado en: Cell Reports Methods, Edición Volume 2, Edición 12, 2022, ISSN 2667-2375
Editor: Cell Press
DOI: 10.1016/j.crmeth.2022.100367

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

Autores: 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
Publicado en: Science, Edición 376, 2024, ISSN 0036-8075
Editor: 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

Autores: Mark Tomishima, Agnete Kirkeby
Publicado en: Journal of Parkinsons Disease, 2021, ISSN 1877-7171
Editor: IOS Press
DOI: 10.3233/jpd-212685

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

Autores: 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
Publicado en: Science, Edición 372/6542, 2021, Página(s) eabf5759, ISSN 0036-8075
Editor: 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.

Autores: Shamma Qarin; Sarah Howlett; Joanne L. Jones; Roger A. Barker
Publicado en: Neuronal Signaling, Edición 5 (3), 2021, ISSN 2059-6553
Editor: Portland Press
DOI: 10.1042/ns20200083

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

Autores: Roger A. Barker, Anders Björklund, Malin Parmar
Publicado en: BioEssays, Edición 46, 2024, ISSN 0265-9247
Editor: 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

Autores: 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
Publicado en: Nature Communications, Edición 15, 2025, ISSN 2041-1723
Editor: Nature Publishing Group
DOI: 10.1038/s41467-024-50877-x

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

Autores: 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
Publicado en: Brain, Edición 144, 2021, Página(s) 3175-3190, ISSN 0006-8950
Editor: Oxford University Press
DOI: 10.1093/brain/awab186

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

Autores: 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
Publicado en: Mol Ther Methods Clin Dev, 2023, ISSN 2329-0501
Editor: 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

Autores: 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
Publicado en: Stem Cell Research & Therapy, Edición 15, 2024, Página(s) 01-06, ISSN 1757-6512
Editor: BioMed Central
DOI: 10.1186/s13287-024-04110-7

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

Autores: Elena Cattaneo, Roger A Barker
Publicado en: Clinical and Translational Medicine, Edición 14, 2024, ISSN 2001-1326
Editor: Wiley Online Library
DOI: 10.1002/ctm2.1746

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

Autores: 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
Publicado en: Advanced Science, Edición Volume 9, Edición 16, 2021, ISSN 2198-3844
Editor: Wiley-VCH
DOI: 10.1002/advs.202104979

Identifying secreted biomarkers of dopaminergic ventral midbrain progenitor cells

Autores: Pedro Rifes, Marc Isaksson, Charlotte Rusimbi, Adrián Ramón Santonja, Jenny Nelander, Thomas Laurell, Agnete Kirkeby
Publicado en: Stem Cell Research and Therapy, 2023, ISSN 1757-6512
Editor: BioMed Central
DOI: 10.1186/s13287-023-03580-5

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

Autores: Pia Rivetti di Val Cervo; Dario Besusso; Paola Conforti; Elena Cattaneo
Publicado en: Nature Reviews. Neurology, Edición 17, 2021, Página(s) 381–392, ISSN 1759-4758
Editor: 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

Autores: 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
Publicado en: Stem Cell Res Ther, Edición Jul 28;14(1), 2023, Página(s) Article number: 189, ISSN 1757-6512
Editor: BioMed Central
DOI: 10.1186/s13287-023-03422-4

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

Autores: 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
Publicado en: Nature Neuroscience, Edición 27, 2024, Página(s) 1260-1273, ISSN 1097-6256
Editor: Nature Publishing Group
DOI: 10.1038/s41593-024-01677-5

Molecular diversity of diencephalic astrocytes reveals adult astrogenesis regulated by Smad4

Autores: 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
Publicado en: EMBO J, Edición 40(21), 2021, Página(s) e107532, ISSN 1460-2075
Editor: EMBO Press
DOI: 10.15252/embj.2020107532

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

Autores: 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
Publicado en: Nature Methods, 2023, ISSN 1548-7091
Editor: Nature Publishing Group
DOI: 10.1038/s41592-023-02080-x

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

Autores: Elena Cattaneo, Dario Besusso
Publicado en: Pharmacological Research, Edición 212, 2025, Página(s) 107569, ISSN 1043-6618
Editor: Academic Press
DOI: 10.1016/j.phrs.2024.107569

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

Autores: Ernest Arenas
Publicado en: Nature, Edición 582/7813, 2020, Página(s) 489-490, ISSN 0028-0836
Editor: Nature Publishing Group
DOI: 10.1038/d41586-020-01817-4

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

Autores: 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
Publicado en: BioRxiv, 2025
Editor: Cold Spring Harbor Laboratory
DOI: 10.1101/2025.01.31.635250

APCDD1 outperforms other dopaminergic cell surface markers in benchmarking study

Autores: 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
Publicado en: Research Square, 2023, ISSN 2693-5015
Editor: Research Square Platform LLC
DOI: 10.21203/rs.3.rs-3266974/v1

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

Autores: 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
Publicado en: BioRxiv, 2024
Editor: 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

Autores: Annabel J Curle, Shaline V Fazal, Shamma Qarin, Sarah K Howlett, Xiaoling He, Roger A Barker, Joanne L Jones
Publicado en: biorxiv, 2024, ISSN 2692-8205
Editor: 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

Autores: 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
Publicado en: BioRxiv, 2024
Editor: Cold Spring Harbor Laboratory
DOI: 10.1101/2024.05.08.593131

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

Autores: Yiling Li, Anthi C. Krontira, Franziska Vierl, Maria L. Richter, Weixu Wang, Juliane Merl-Pham, Fabian J. Theis, Stefanie M. Hauck, Magdalena Götz
Publicado en: BioRxiv, 2025
Editor: Cold Spring Harbor Laboratory
DOI: 10.1101/2025.02.13.635953

Generation of human cerebral organoids with a structured outer subventricular zone

Autores: 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
Publicado en: bioRxiv, 2023, ISSN 2692-8205
Editor: Cold Spring Harbor Laboratory
DOI: 10.1101/2023.02.17.528906

Scaling of cell and gene therapies to population

Autores: Sebastian Knöbel, Andreas Bosio
Publicado en: Handbook of Clinical Neurology, Cell and Gene Therapies for Neurologic Diseases, 2024, Página(s) 145-154
Editor: Elsevier
DOI: 10.1016/b978-0-323-90120-8.00012-5

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