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

Project Information

RECOMB

Grant agreement ID: 755170

DOI

10.3030/755170

Project closed

EC signature date 21 November 2017

Start date 1 January 2018

End date 31 December 2024

Funded under

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

Total cost

€ 5 990 460,75

EU contribution

€ 5 990 460,00

Coordinated by

ACADEMISCH ZIEKENHUIS LEIDEN
Netherlands

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

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

Publications

Peer reviewed articles (25)

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

Author(s): 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
Published in: Volume 11, Issue 1, 2021, ISSN 2075-4426
Publisher: mdpi
DOI: 10.3390/jpm11030221

Utilizing epigenetic regulators to improve HSC-based lentiviral gene therapy

Author(s): 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
Published in: Blood Advances, Issue 8, 2024, Page(s) 4936-4947, ISSN 2473-9529
Publisher: Blood advances
DOI: 10.1182/bloodadvances.2024013047

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

Author(s): Veronica Della Chiara, Lucia Daxinger, Frank J T Staal
Published in: Cells, 2021, ISSN 2073-4409
Publisher: mdpi
DOI: 10.3390/cells10051074

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

Author(s): 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
Published in: VOLUME=23;ISSUE=21;TITLE=International Journal of Molecular Sciences, Issue 3, 2022, ISSN 1422-0067
Publisher: Multidisciplinary Digital Publishing Institute (MDPI)
DOI: 10.3390/ijms232112736

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

Author(s): 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
Published in: Molecular Therapy - Methods & Clinical Development, Issue 17, 2020, Page(s) 666-682, ISSN 2329-0501
Publisher: 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

Author(s): 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
Published in: Biomedicines, Issue 12, 2024, Page(s) 1495, ISSN 2227-9059
Publisher: 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

Author(s): Michael A. Morgan, Hildegard Büning, Martin Sauer, Axel Schambach
Published in: Frontiers in Immunology, Issue 11, 2020, ISSN 1664-3224
Publisher: Frontiers in immunology
DOI: 10.3389/fimmu.2020.01965

Inflammation and Wnt Signaling: Target for Immunomodulatory Therapy?

Author(s): Imen Jridi, Kirsten Canté-Barrett, Karin Pike-Overzet, Frank J. T. Staal
Published in: Frontiers in Cell and Developmental Biology, Issue 8, 2021, ISSN 2296-634X
Publisher: frontiers
DOI: 10.3389/fcell.2020.615131

HyperIgE in hypomorphic recombination-activating gene defects

Author(s): Maria Carmina Castiello, Chiara Brandas, Valentina Capo, Anna Villa
Published in: Current Opinion in Immunology, Issue 80, 2023, Page(s) 102279, ISSN 0952-7915
Publisher: Elsevier BV
DOI: 10.1016/j.coi.2022.102279

Gene therapy strategies for RAG1 deficiency: Challenges and breakthroughs

Author(s): Giorgio Gilioli, Arjan C. Lankester, Sander de Kivit, Frank J.T. Staal, Lisa M. Ott de Bruin
Published in: Immunology Letters, Issue 270, 2024, Page(s) 106931, ISSN 0165-2478
Publisher: Elsevier BV
DOI: 10.1016/j.imlet.2024.106931

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

Author(s): Cordes M, Pike-Overzet K, Van Den Akker EB, Staal FJT, Canté-Barrett K.
Published in: Frontiers in Cell and Developmental Biology, 2023, ISSN 2296-634X
Publisher: Frontiers Media S.A.
DOI: 10.3389/fcell.2023.1163529

Advances in gene therapy for inborn errors of immunity

Author(s): Lisa M. Ott de Bruin, Arjan C. Lankester, Frank J.T. Staal
Published in: Current Opinion in Allergy & Clinical Immunology, Issue 23, 2024, Page(s) 467-477, ISSN 1528-4050
Publisher: Lippincott Williams & Wilkins Ltd.
DOI: 10.1097/aci.0000000000000952

Cell intrinsic regulation of external hematopoietic stem cell stress

Author(s): Frank J. T. Staal
Published in: Stem Cell Investigation, Issue 5, 2018, Page(s) 16-16, ISSN 2313-0792
Publisher: 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.

Author(s): 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
Published in: Science immunology, 2022, ISSN 2470-9468
Publisher: American Association for the Advancement of Science
DOI: 10.1126/sciimmunol.ade0182

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

Author(s): 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
Published in: Frontiers in Genome Editing, Issue 2, 2021, ISSN 2673-3439
Publisher: 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

Author(s): Laura Garcia-Perez, Marja C.J.A. van Eggermond, Elisa Maietta, Marie-Louise P. van der Hoorn, Karin Pike-Overzet, Frank J. T. Staal
Published in: Frontiers in Immunology, Issue 11, 2021, ISSN 1664-3224
Publisher: 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.

Author(s): Kirsten Canté-Barrett; Frank J.T. Staal
Published in: EBioMedicine, Issue 2, 2020, Page(s) 102961, ISSN 0140-6736
Publisher: The Lancet Publishing Group
DOI: 10.1016/j.ebiom.2020.103015

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

Author(s): Braams M, Pike-Overzet K, Staal FJT.
Published in: Frontiers in immunology, 2023, ISSN 1664-3224
Publisher: Frontiers Media S.A.
DOI: 10.3389/fimmu.2023.1210818

Innovative Cell-Based Therapies and Conditioning to Cure RAG Deficiency

Author(s): Anna Villa, Valentina Capo, Maria Carmina Castiello
Published in: Frontiers in Immunology, Issue 11, 2020, ISSN 1664-3224
Publisher: frontiers
DOI: 10.3389/fimmu.2020.607926

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

Author(s): 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
Published in: Frontiers in Immunology, Issue 14, 2023, ISSN 1664-3224
Publisher: 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

Author(s): 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
Published in: Stem Cell Reports, Issue 13/4, 2019, Page(s) 590-598, ISSN 2213-6711
Publisher: 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.

Author(s): Bastani S, Staal FJT, Canté-Barrett K.
Published in: Stem Cell Investig, 2023, ISSN 2306-9759
Publisher: AME Publishing Company
DOI: 10.21037/sci-2023-016

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

Author(s): Hidde A. Zittersteijn, Cornelis L. Harteveld, Stefanie Klaver-Flores, Arjan C. Lankester, Rob C. Hoeben, Frank J. T. Staal, Manuel A. F. V. Gonçalves
Published in: Frontiers in Genome Editing, Issue 2, 2021, ISSN 2673-3439
Publisher: 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

Author(s): Laura Garcia-Perez, Anita Ordas, Kirsten Canté-Barrett, Pauline Meij, Karin Pike-Overzet, Arjan Lankester, Frank J. T. Staal
Published in: Pharmaceutics, Issue 12/6, 2020, Page(s) 549, ISSN 1999-4923
Publisher: Multidisciplinary Digital Publishing Institute (MDPI)
DOI: 10.3390/pharmaceutics12060549

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

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

Conference proceedings (4)

SIDUS® technology for viral vector manufacturing

Author(s): Alfred Luitjens
Published in: 2019
Publisher: ASGCT

Manufacturing Lentiviral Vector as Gene Therapy for RAG1-SCID

Author(s): Alfred Luitjens
Published in: PepTalk 2020: The Protein Science Week. Proceedings of a meeting held 20-24 January 2020, San Diego, California, USA. Abstracts, 2020, ISBN 9781713809784
Publisher: Cambridge EnerTech

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

Author(s): 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
Published in: Human Gene Therapy, 2021
Publisher: 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

Author(s): 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
Published in: Molecular Therapy, 2021, ISSN 1525-0016
Publisher: Cell Press

Non-peer reviewed articles (1)

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

Author(s): 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.
Published in: molecular therapy, 2021, ISSN 1525-0016
Publisher: Nature Publishing Group
DOI: 10.1016/j.ymthe.2021.06.017