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

Deliverables

RECOMB Website

Setting up of RECOMB project dedicated website (M9)

Publications

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, 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, 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, 8, 2021, ISSN 2296-634X
Publisher: frontiers
DOI: 10.3389/fcell.2020.615131

Cell intrinsic regulation of external hematopoietic stem cell stress

Author(s): Frank J. T. Staal
Published in: Stem Cell Investigation, 5, 2018, Page(s) 16-16, ISSN 2313-0792
Publisher: AME publishing company
DOI: 10.21037/sci.2018.05.01

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, 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, 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, 2, 2020, Page(s) 102961, ISSN 0140-6736
Publisher: The Lancet Publishing Group
DOI: 10.1016/j.ebiom.2020.103015

Innovative Cell-Based Therapies and Conditioning to Cure RAG Deficiency

Author(s): Anna Villa, Valentina Capo, Maria Carmina Castiello
Published in: Frontiers in Immunology, 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, 13/4, 2019, Page(s) 590-598, ISSN 2213-6711
Publisher: Cell Press
DOI: 10.1016/j.stemcr.2019.08.008

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, 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, 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, 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