Deliverables Documents, reports (11) Efficacy of Artemis clinical batch and ODD status Efficacy of Artemis clinical batch and ODD status Implementation of CliniMACS Prodigy in clinical trial Implementation of CliniMACS Prodigy in clinical trial Correct OS in mouse model and patient cells Correct OS in mouse model and patient cells A set of clinically applicable vectors that can correct RAG1 and RAG2 deficiency in mouse models and human cells A set of clinically applicable vectors that can correct RAG1 and RAG2 deficiency in mouse models and human cells Gene editing protocol for one disease entity Gene editing protocol for one disease entity Website for SCIDNET Website for SCIDNET Minutes of 1st General Assembly meeting Minutes of 1st General Assembly meeting Minutes of first SAB Minutes of first SAB Minutes of third SAB Minutes of third SAB Minutes of second SAB Minutes of second SAB Project kick-off meeting minutes Project kick-off meeting minutes Demonstrators, pilots, prototypes (2) Initiation of clinical trial Initiation of clinical trial Setting up of SCIDNET project website Setting up of SCIDNET project website Publications Peer reviewed articles (16) Efficacy of lentivirus-mediated gene therapy in an Omenn syndrome recombination-activating gene 2 mouse model is not hindered by inflammation and immune dysregulation Author(s): Valentina Capo, Maria Carmina Castiello, Elena Fontana, Sara Penna, Marita Bosticardo, Elena Draghici, Luigi P. Poliani, Lucia Sergi Sergi, Rosita Rigoni, Barbara Cassani, Monica Zanussi, Paola Carrera, Paolo Uva, Kerry Dobbs, Nicolò Sacchetti, Luigi D. Notarangelo, Niek P. van Til, Gerard Wagemaker, Anna Villa Published in: Journal of Allergy and Clinical Immunology, Issue 142/3, 2018, Page(s) 928-941.e8, ISSN 0091-6749 Publisher: Mosby Inc. DOI: 10.1016/j.jaci.2017.11.015 Generation of adult human T-cell progenitors for immunotherapeutic applications Author(s): Laura Simons, Kuiying Ma, Corinne de Chappedelaine, Ranjita Devi Moiranghtem, Elodie Elkaim, Juliette Olivré, Sandrine Susini, Kevin Appourchaux, Christian Reimann, Hanem Sadek, Olivier Pellé, Nicolas Cagnard, Elisa Magrin, Chantal Lagresle-Peyrou, Tom Taghon, Antonio Rausell, Marina Cavazzana, Isabelle André-Schmutz Published in: Journal of Allergy and Clinical Immunology, Issue 141/4, 2018, Page(s) 1491-1494.e4, ISSN 0091-6749 Publisher: Mosby Inc. DOI: 10.1016/j.jaci.2017.10.034 Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements Author(s): Annalisa Lattanzi, Vasco Meneghini, Giulia Pavani, Fatima Amor, Sophie Ramadier, Tristan Felix, Chiara Antoniani, Cecile Masson, Olivier Alibeu, Ciaran Lee, Matthew H. Porteus, Gang Bao, Mario Amendola, Fulvio Mavilio, Annarita Miccio Published in: Molecular Therapy, Issue 27/1, 2019, Page(s) 137-150, ISSN 1525-0016 Publisher: Nature Publishing Group DOI: 10.1016/j.ymthe.2018.10.008 Preclinical Development of a Lentiviral Vector for Gene Therapy of X-Linked Severe Combined Immunodeficiency Author(s): Valentina Poletti, Sabine Charrier, Guillaume Corre, Bernard Gjata, Alban Vignaud, Fang Zhang, Michael Rothe, Axel Schambach, H. Bobby Gaspar, Adrian J. Thrasher, Fulvio Mavilio Published in: Molecular Therapy - Methods & Clinical Development, Issue 9, 2018, Page(s) 257-269, ISSN 2329-0501 Publisher: Elsevier DOI: 10.1016/j.omtm.2018.03.002 Autologous Stem-Cell-Based Gene Therapy for Inherited Disorders: State of the Art and Perspectives Author(s): Frank J. T. Staal, Alessandro Aiuti, Marina Cavazzana Published in: Frontiers in Pediatrics, Issue 7, 2019, ISSN 2296-2360 Publisher: Frontiers in Pediatrics DOI: 10.3389/fped.2019.00443 Concise Review: BOOSTING T Cell Reconstitution Following Allogeneic Transplantation—Current Concepts and Future Perspectives Author(s): Laura Simons, Marina Cavazzana, Isabelle André Published in: STEM CELLS Translational Medicine, 2019, ISSN 2157-6564 Publisher: AlphaMed Press DOI: 10.1002/sctm.18-0248 Baboon envelope LVs efficiently transduced human adult, fetal, and progenitor T cells and corrected SCID-X1 T-cell deficiency Author(s): Ornellie Bernadin, Fouzia Amirache, Anais Girard-Gagnepain, Ranjita Devi Moirangthem, Camille Lévy, Kuiying Ma, Caroline Costa, Didier Nègre, Christian Reimann, David Fenard, Agata Cieslak, Vahid Asnafi, Hanem Sadek, Rana Mhaidly, Marina Cavazzana, Chantal Lagresle-Peyrou, François-Loïc Cosset, Isabelle André, Els Verhoeyen Published in: Blood Advances, Issue 3/3, 2019, Page(s) 461-475, ISSN 2473-9529 Publisher: Blood DOI: 10.1182/bloodadvances.2018027508 Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID Author(s): Sabine Charrier, Chantal Lagresle-Peyrou, Valentina Poletti, Michael Rothe, Grégory Cédrone, Bernard Gjata, Fulvio Mavilio, Alain Fischer, Axel Schambach, Jean-Pierre de Villartay, Marina Cavazzana, Salima Hacein-Bey-Abina, Anne Galy Published in: Molecular Therapy - Methods & Clinical Development, Issue 15, 2019, Page(s) 232-245, ISSN 2329-0501 Publisher: lsevier DOI: 10.1016/j.omtm.2019.08.014 Enhancing Lentiviral and Alpharetroviral Transduction of Human Hematopoietic Stem Cells for Clinical Application Author(s): Juliane W. Schott, Diego León-Rico, Carolina B. Ferreira, Karen F. Buckland, Giorgia Santilli, Myriam A. Armant, Axel Schambach, Alessia Cavazza, Adrian J. Thrasher Published in: Molecular Therapy - Methods & Clinical Development, Issue 14, 2019, Page(s) 134-147, ISSN 2329-0501 Publisher: Elsevier DOI: 10.1016/j.omtm.2019.05.015 ESGCT 27th Annual Congress In collaboration with SETGyc Barcelona, Spain October 22–25, 2019 Abstracts Author(s): M Zahn, C Lulay, S Afzal, R Fronza, W Wang, R Gabriel, B Gaspar, M Schmidt, I Gil-Farina Published in: Human Gene Therapy, Issue 30/11, 2019, Page(s) A1-A221, ISSN 1043-0342 Publisher: Mary Ann Liebert Inc. DOI: 10.1089/hum.2019.29095.abstracts Refining strategies to translate genome editing to the clinic Author(s): Tatjana I Cornu, Claudio Mussolino, Toni Cathomen Published in: Nature Medicine, Issue 23/4, 2017, Page(s) 415-423, ISSN 1078-8956 Publisher: Nature Publishing Group DOI: 10.1038/nm.4313 Therapeutic genome editing with engineered nucleases Author(s): S. A. Haas, V. Dettmer, T. Cathomen Published in: Hämostaseologie, Issue 37/1, 2017, Page(s) 45-52, ISSN 0720-9355 Publisher: F. K. Schattauer Verlag DOI: 10.5482/HAMO-16-09-0035 Multimodal Lentiviral Vectors for Pharmacologically Controlled Switching Between Constitutive Single Gene Expression and Tetracycline-Regulated Multiple Gene Collaboration Author(s): Maike Stahlhut, Axel Schambach, Olga S. Kustikova Published in: Human Gene Therapy Methods, Issue 28/4, 2017, Page(s) 191-204, ISSN 1946-6536 Publisher: Mary Ann Liebert Inc. DOI: 10.1089/hgtb.2017.073 An optimized lentiviral vector system for conditional RNAi and efficient cloning of microRNA embedded short hairpin RNA libraries Author(s): Felix F. Adams, Dirk Heckl, Thomas Hoffmann, Steven R. Talbot, Arnold Kloos, Felicitas Thol, Michael Heuser, Johannes Zuber, Axel Schambach, Adrian Schwarzer Published in: Biomaterials, Issue 139, 2017, Page(s) 102-115, ISSN 0142-9612 Publisher: Pergamon Press Ltd. DOI: 10.1016/j.biomaterials.2017.05.032 Viral and Synthetic RNA Vector Technologies and Applications Author(s): Juliane W Schott, Michael Morgan, Melanie Galla, Axel Schambach Published in: Molecular Therapy, Issue 24/9, 2016, Page(s) 1513-1527, ISSN 1525-0016 Publisher: Nature Publishing Group DOI: 10.1038/mt.2016.143 Alpharetroviral self-inactivating vectors produced by a superinfection-resistant stable packaging cell line allow genetic modification of primary human T lymphocytes Author(s): Verena Labenski, Julia D. Suerth, Elke Barczak, Dirk Heckl, Camille Levy, Ornellie Bernadin, Emmanuelle Charpentier, David A. Williams, Boris Fehse, Els Verhoeyen, Axel Schambach Published in: Biomaterials, Issue 97, 2016, Page(s) 97-109, ISSN 0142-9612 Publisher: Pergamon Press Ltd. DOI: 10.1016/j.biomaterials.2016.04.019 Conference proceedings (2) Using the CliniMACS PRODIGY® for CD34 enrichment and transduction of mobilised peripheral blood stem cells (mPBSC) Author(s): E Armenteros-Monterroso1,2, K F Buckland2,1, A Diasakou1,2, I Pereira2,1, D Leon-Rico2,1, S Reinartz3, D Krenz3, U Bissels3, I Johnston3, E Papanikolaou3, C Booth1,2, A J Thrasher A J2,1 Published in: HUMAN GENE THERAPY, Issue Volume: 30 Issue: 11, 2019, Page(s) A89, ISSN 1043-0342 Publisher: Mary Ann Liebert Inc Manufacture of an ATMP for the treatment of X-linked Severe Combined Immunodeficiency (X-SCID) Author(s): Leon-Rico D, Schott JW, Armenteros-Monterroso E, Buckland KF, Diasakou A, Pereira I, Ferreira CB, Cavazza A, Shaw KL, Armant M, Kohn DB, Pai SY, Thasher AJ, Booth C Published in: HUMAN GENE THERAPY, Issue Volume: 30 Issue: 11, 2019, Page(s) A86, ISSN 1043-0342 Publisher: Mary Ann Liebert, Inc Searching for OpenAIRE data... 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