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

Deliverables

Setting up of RECOMB project dedicated website M9

Publications

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

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

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

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

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

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

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

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

Deliverables

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