Skip to main content

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

Deliverables

RECOMB Website

Setting up of RECOMB project dedicated website (M9)

Searching for OpenAIRE data...

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