Stem-cell based gene therapy for recombination deficient SCID (RECOMB)

Información del proyecto

RECOMB

Identificador del acuerdo de subvención: 755170

DOI

10.3030/755170

Proyecto cerrado

Fecha de la firma de la CE 21 Noviembre 2017

Fecha de inicio 1 Enero 2018

Fecha de finalización 31 Diciembre 2024

Financiado con arreglo a

• SOCIETAL CHALLENGES - Health, demographic change and well-being

Coste total

€ 5 990 460,75

Aportación de la UE

€ 5 990 460,00

Coordinado por

ACADEMISCH ZIEKENHUIS LEIDEN
Netherlands

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

Open Research Data Pilot (1)

D11.3

ORDP: Open Research Data Pilot. Drfat data availbale

Websites, patent fillings, videos etc. (1)

RECOMB Website

Setting up of RECOMB project dedicated website M9

Publicaciones

Peer reviewed articles (25)

Genetic Correction of IL-10RB Deficiency Reconstitutes Anti-Inflammatory Regulation in iPSC-Derived Macrophages.

Autores: Dirk Hoffmann; Johanna Sens; Sebastian Brennig; Daniel Brand; Friederike Philipp; Philippe Vollmer Barbosa; Philippe Vollmer Barbosa; Johannes Kuehle; Doris Steinemann; Daniela Lenz; Theresa Buchegger; Michael A. Morgan; Christine S. Falk; Christoph Klein; Nico Lachmann; Axel Schambach; Axel Schambach
Publicado en: Volume 11, Edición 1, 2021, ISSN 2075-4426
Editor: mdpi
DOI: 10.3390/jpm11030221

Utilizing epigenetic regulators to improve HSC-based lentiviral gene therapy

Autores: Parisa Tajer, Emin Onur Karakaslar, Kirsten Canté-Barrett, Brigitta A. E. Naber, Sandra A. Vloemans, Marja C. J. A. van Eggermond, Marie-Louise van der Hoorn, Erik van den Akker, Karin Pike-Overzet, Frank J. T. Staal
Publicado en: Blood Advances, Edición 8, 2024, Página(s) 4936-4947, ISSN 2473-9529
Editor: Blood advances
DOI: 10.1182/bloodadvances.2024013047

The Route of Early T Cell Development: Crosstalk between Epigenetic and Transcription Factors

Autores: Veronica Della Chiara, Lucia Daxinger, Frank J T Staal
Publicado en: Cells, 2021, ISSN 2073-4409
Editor: mdpi
DOI: 10.3390/cells10051074

IL3 has a detrimental effect on hematopoietic stem cell self-renewal in transplantation settings

Autores: Parisa Tajer; Kirsten Canté-Barrett; Brigitta A. E. Naber; Sandra A. Vloemans; Marja C. J. A. van Eggermond; Marie-Louise van der Hoorn; Karin Pike-Overzet; Frank J. T. Staal
Publicado en: VOLUME=23;ISSUE=21;TITLE=International Journal of Molecular Sciences, Edición 3, 2022, ISSN 1422-0067
Editor: Multidisciplinary Digital Publishing Institute (MDPI)
DOI: 10.3390/ijms232112736

Successful Preclinical Development of Gene Therapy for Recombinase-Activating Gene-1-Deficient SCID

Autores: Laura Garcia-Perez, Marja van Eggermond, Lieke van Roon, Sandra A. Vloemans, Martijn Cordes, Axel Schambach, Michael Rothe, Dagmar Berghuis, Chantal Lagresle-Peyrou, Marina Cavazzana, Fang Zhang, Adrian J. Thrasher, Daniela Salvatori, Pauline Meij, Anna Villa, Jacques J.M. Van Dongen, Jaap-Jan Zwaginga, Mirjam van der Burg, H. Bobby Gaspar, Arjan Lankester, Frank J.T. Staal, Karin Pike-Overzet
Publicado en: Molecular Therapy - Methods & Clinical Development, Edición 17, 2020, Página(s) 666-682, ISSN 2329-0501
Editor: Cell press
DOI: 10.1016/j.omtm.2020.03.016

Restoration of T and B Cell Differentiation after RAG1 Gene Transfer in Human RAG1 Defective Hematopoietic Stem Cells

Autores: Nataël Sorel, Francisco Díaz-Pascual, Boris Bessot, Hanem Sadek, Chloé Mollet, Myriam Chouteau, Marco Zahn, Irene Gil-Farina, Parisa Tajer, Marja van Eggermond, Dagmar Berghuis, Arjan C. Lankester, Isabelle André, Richard Gabriel, Marina Cavazzana, Kasrin Pike-Overzet, Frank J. T. Staal, Chantal Lagresle-Peyrou
Publicado en: Biomedicines, Edición 12, 2024, Página(s) 1495, ISSN 2227-9059
Editor: MDPI Biomedicines
DOI: 10.3390/biomedicines12071495

Use of Cell and Genome Modification Technologies to Generate Improved “Off-the-Shelf” CAR T and CAR NK Cells

Autores: Michael A. Morgan, Hildegard Büning, Martin Sauer, Axel Schambach
Publicado en: Frontiers in Immunology, Edición 11, 2020, ISSN 1664-3224
Editor: Frontiers in immunology
DOI: 10.3389/fimmu.2020.01965

Inflammation and Wnt Signaling: Target for Immunomodulatory Therapy?

Autores: Imen Jridi, Kirsten Canté-Barrett, Karin Pike-Overzet, Frank J. T. Staal
Publicado en: Frontiers in Cell and Developmental Biology, Edición 8, 2021, ISSN 2296-634X
Editor: frontiers
DOI: 10.3389/fcell.2020.615131

HyperIgE in hypomorphic recombination-activating gene defects

Autores: Maria Carmina Castiello, Chiara Brandas, Valentina Capo, Anna Villa
Publicado en: Current Opinion in Immunology, Edición 80, 2023, Página(s) 102279, ISSN 0952-7915
Editor: Elsevier BV
DOI: 10.1016/j.coi.2022.102279

Gene therapy strategies for RAG1 deficiency: Challenges and breakthroughs

Autores: Giorgio Gilioli, Arjan C. Lankester, Sander de Kivit, Frank J.T. Staal, Lisa M. Ott de Bruin
Publicado en: Immunology Letters, Edición 270, 2024, Página(s) 106931, ISSN 0165-2478
Editor: Elsevier BV
DOI: 10.1016/j.imlet.2024.106931

Multi-omic analyses in immune cell development with lessons learned from T cell development.

Autores: Cordes M, Pike-Overzet K, Van Den Akker EB, Staal FJT, Canté-Barrett K.
Publicado en: Frontiers in Cell and Developmental Biology, 2023, ISSN 2296-634X
Editor: Frontiers Media S.A.
DOI: 10.3389/fcell.2023.1163529

Advances in gene therapy for inborn errors of immunity

Autores: Lisa M. Ott de Bruin, Arjan C. Lankester, Frank J.T. Staal
Publicado en: Current Opinion in Allergy & Clinical Immunology, Edición 23, 2024, Página(s) 467-477, ISSN 1528-4050
Editor: Lippincott Williams & Wilkins Ltd.
DOI: 10.1097/aci.0000000000000952

Cell intrinsic regulation of external hematopoietic stem cell stress

Autores: Frank J. T. Staal
Publicado en: Stem Cell Investigation, Edición 5, 2018, Página(s) 16-16, ISSN 2313-0792
Editor: AME publishing company
DOI: 10.21037/sci.2018.05.01

Single-cell immune profiling reveals thymus-seeding populations, T cell commitment, and multilineage development in the human thymus.

Autores: Cordes M, Canté-Barrett K, van den Akker EB, Moretti FA, Kiełbasa SM, Vloemans SA, Garcia-Perez L, Teodosio C, van Dongen JJM, Pike-Overzet K, Reinders MJT, Staal FJT
Publicado en: Science immunology, 2022, ISSN 2470-9468
Editor: American Association for the Advancement of Science
DOI: 10.1126/sciimmunol.ade0182

Genomic Engineering in Human Hematopoietic Stem Cells: Hype or Hope?

Autores: Stefanie Klaver-Flores, Hidde A. Zittersteijn, Kirsten Canté-Barrett, Arjan Lankester, Rob C. Hoeben, Manuel A. F. V. Gonçalves, Karin Pike-Overzet, Frank J. T. Staal
Publicado en: Frontiers in Genome Editing, Edición 2, 2021, ISSN 2673-3439
Editor: frontiers
DOI: 10.3389/fgeed.2020.615619

A Novel Branched DNA-Based Flowcytometric Method for Single-Cell Characterization of Gene Therapy Products and Expression of Therapeutic Genes

Autores: Laura Garcia-Perez, Marja C.J.A. van Eggermond, Elisa Maietta, Marie-Louise P. van der Hoorn, Karin Pike-Overzet, Frank J. T. Staal
Publicado en: Frontiers in Immunology, Edición 11, 2021, ISSN 1664-3224
Editor: frontiers
DOI: 10.3389/fimmu.2020.607991

An adequate human T cell repertoire from a single T cell progenitor: Lessons from an experiment of nature.

Autores: Kirsten Canté-Barrett; Frank J.T. Staal
Publicado en: EBioMedicine, Edición 2, 2020, Página(s) 102961, ISSN 0140-6736
Editor: The Lancet Publishing Group
DOI: 10.1016/j.ebiom.2020.103015

The recombinase activating genes: architects of immune diversity during lymphocyte development.

Autores: Braams M, Pike-Overzet K, Staal FJT.
Publicado en: Frontiers in immunology, 2023, ISSN 1664-3224
Editor: Frontiers Media S.A.
DOI: 10.3389/fimmu.2023.1210818

Innovative Cell-Based Therapies and Conditioning to Cure RAG Deficiency

Autores: Anna Villa, Valentina Capo, Maria Carmina Castiello
Publicado en: Frontiers in Immunology, Edición 11, 2020, ISSN 1664-3224
Editor: frontiers
DOI: 10.3389/fimmu.2020.607926

Partial correction of immunodeficiency by lentiviral vector gene therapy in mouse models carrying Rag1 hypomorphic mutations

Autores: Maria Carmina Castiello, Martina Di Verniere, Elena Draghici, Elena Fontana, Sara Penna, Lucia Sereni, Alessandra Zecchillo, Denise Minuta, Paolo Uva, Marco Zahn, Irene Gil-Farina, Andrea Annoni, Silvia Iaia, Lisa M. Ott de Bruin, Luigi D. Notarangelo, Karin Pike-Overzet, Frank J. T. Staal, Anna Villa, Valentina Capo
Publicado en: Frontiers in Immunology, Edición 14, 2023, ISSN 1664-3224
Editor: Front Immunol.
DOI: 10.3389/fimmu.2023.1268620

Targeted Repair of p47-CGD in iPSCs by CRISPR/Cas9: Functional Correction without Cleavage in the Highly Homologous Pseudogenes

Autores: Denise Klatt, Erica Cheng, Friederike Philipp, Anton Selich, Julia Dahlke, Reinhold E. Schmidt, Juliane W. Schott, Hildegard Büning, Dirk Hoffmann, Adrian J. Thrasher, Axel Schambach
Publicado en: Stem Cell Reports, Edición 13/4, 2019, Página(s) 590-598, ISSN 2213-6711
Editor: Cell Press
DOI: 10.1016/j.stemcr.2019.08.008

The quest for the holy grail: overcoming challenges in expanding human hematopoietic stem cells for clinical use.

Autores: Bastani S, Staal FJT, Canté-Barrett K.
Publicado en: Stem Cell Investig, 2023, ISSN 2306-9759
Editor: AME Publishing Company
DOI: 10.21037/sci-2023-016

A Small Key for a Heavy Door: Genetic Therapies for the Treatment of Hemoglobinopathies

Autores: Hidde A. Zittersteijn, Cornelis L. Harteveld, Stefanie Klaver-Flores, Arjan C. Lankester, Rob C. Hoeben, Frank J. T. Staal, Manuel A. F. V. Gonçalves
Publicado en: Frontiers in Genome Editing, Edición 2, 2021, ISSN 2673-3439
Editor: frontiers
DOI: 10.3389/fgeed.2020.617780

Preclinical Development of Autologous Hematopoietic Stem Cell-Based Gene Therapy for Immune Deficiencies: A Journey from Mouse Cage to Bed Side

Autores: Laura Garcia-Perez, Anita Ordas, Kirsten Canté-Barrett, Pauline Meij, Karin Pike-Overzet, Arjan Lankester, Frank J. T. Staal
Publicado en: Pharmaceutics, Edición 12/6, 2020, Página(s) 549, ISSN 1999-4923
Editor: Multidisciplinary Digital Publishing Institute (MDPI)
DOI: 10.3390/pharmaceutics12060549

A Large Cohort of RAG1/2-Deficient SCID Patients—Clinical, Immunological, and Prognostic Analysis

Autores: Noa Greenberg-Kushnir, Yu Nee Lee, Amos J. Simon, Atar Lev, Nufar Marcus, Omar Abuzaitoun, Raz Somech, Tali Stauber
Publicado en: Journal of Clinical Immunology, Edición 40/1, 2020, Página(s) 211-222, ISSN 0271-9142
Editor: Kluwer Academic/Plenum Publishers
DOI: 10.1007/s10875-019-00717-1

Conference proceedings (4)

SIDUS® technology for viral vector manufacturing

Autores: Alfred Luitjens
Publicado en: 2019
Editor: ASGCT

Manufacturing Lentiviral Vector as Gene Therapy for RAG1-SCID

Autores: Alfred Luitjens
Publicado en: PepTalk 2020: The Protein Science Week. Proceedings of a meeting held 20-24 January 2020, San Diego, California, USA. Abstracts, 2020, ISBN 9781713809784
Editor: Cambridge EnerTech

Efficacy assessment of lentiviral vector gene therapy in the immune dysregulated hypomorphic Rag1 mice

Autores: V Capo, M C Castiello, E Draghici, S Penna, M Di Verniere, E Fontana, D Minuta, S Porcellini, P Uva, LD Notarangelo, K Pike-Overzet, FJT Staal, A Villa1
Publicado en: Human Gene Therapy, 2021
Editor: Mary Ann Liebert, Inc.
DOI: 10.1089/hum.2021.29180.abstracts

Lentiviral Vector Gene Therapy in Rag1 Hypomorphic Mice: Treating Immunodeficiency in the Context of Immune Dysregulation

Autores: Valentina Capo, Maria Carmina Castiello, Elena Draghici, Sara Penna, Martina Di Verniere, Elena Fontana, Lucia Sereni, Paolo Uva, Luigi D. Notarangelo, Karin Pike-Overzet, Frank J. T. Staal, Anna Villa
Publicado en: Molecular Therapy, 2021, ISSN 1525-0016
Editor: Cell Press

Non-peer reviewed articles (1)

Predicting genotoxicity of viral vectors for stem cell gene therapy using gene expression-based machine learning

Autores: Schwarzer A, Talbot SR, Selich A, Morgan M, Schott JW, Dittrich-Breiholz O, Bastone AL, Weigel B, Ha TC, Dziadek V, Gijsbers R, Thrasher AJ, Staal FJT, Gaspar HB, Modlich U, Schambach A, Rothe M.
Publicado en: molecular therapy, 2021, ISSN 1525-0016
Editor: Nature Publishing Group
DOI: 10.1016/j.ymthe.2021.06.017