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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)

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, Issue 17, 2020, Page(s) 666-682, ISSN 2329-0501
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
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
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, Issue 5, 2018, Page(s) 16-16, ISSN 2313-0792
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, Issue 2, 2021, ISSN 2673-3439
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
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
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, Issue 11, 2020, ISSN 1664-3224
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
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, Issue 2, 2021, ISSN 2673-3439
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
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
DOI: 10.1007/s10875-019-00717-1

SIDUS® technology for viral vector manufacturing

Author(s): Alfred Luitjens
Published in: 2019