Expanding and extending gene therapy of monogenic diseases of the haematopoietic system

Project Information

GENE FOR CURE

Grant agreement ID: 693762

DOI

10.3030/693762

Project closed

EC signature date 13 December 2016

Start date 1 October 2016

End date 30 September 2024

Funded under

• EXCELLENT SCIENCE - European Research Council (ERC)

Total cost

€ 2 445 267,52

EU contribution

€ 2 445 267,52

Coordinated by

INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE
France

CORDIS provides links to public deliverables and publications of HORIZON projects.

Links to deliverables and publications from FP7 projects, as well as links to some specific result types such as dataset and software, are dynamically retrieved from OpenAIRE .

Publications

Peer reviewed articles (11)

Innovations Needed for Effective Implementation of Ex Vivo Gene Therapies

Author(s): Marina Cavazzana
Published in: Frontiers in Medicine, Issue 4, 2017, ISSN 2296-858X
Publisher: Frontiers Media SA
DOI: 10.3389/fmed.2017.00029

Gene therapy targeting haematopoietic stem cells for inherited diseases: progress and challenges

Author(s): Marina Cavazzana, Frederic D. Bushman, Annarita Miccio, Isabelle André-Schmutz, Emmanuelle Six
Published in: Nature Reviews Drug Discovery, Issue 18, 2023, Page(s) 447-462, ISSN 1474-1776
Publisher: Nature Publishing Group
DOI: 10.1038/s41573-019-0020-9

A combination of cyclophosphamide and interleukin-2 allows CD4+ T cells converted to Tregs to control <i>scurfy</i> syndrome

Author(s): Marianne Delville, Florence Bellier, Juliette Leon, Roman Klifa, Sabrina Lizot, Hélène Vinçon, Steicy Sobrino, Romane Thouenon, Armance Marchal, Alexandrine Garrigue, Juliette Olivré, Soëli Charbonnier, Chantal Lagresle-Peyrou, Mario Amendola, Axel Schambach, David Gross, Baptiste Lamarthée, Christophe Benoist, Julien Zuber, Isabelle André, Marina Cavazzana, Emmanuelle Six
Published in: Blood, Issue 137, 2021, Page(s) 2326-2336, ISSN 0006-4971
Publisher: American Society of Hematology
DOI: 10.1182/blood.2020009187

Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome

Author(s): A. Magnani, M. Semeraro, F. Adam, C. Booth, L. Dupré, E. C. Morris, A. Gabrion, C. Roudaut, D. Borgel, A. Toubert, E. Clave, C. Abdo, G. Gorochov, R. Petermann, M. Guiot, M. Miyara, D. Moshous, E. Magrin, A. Denis, F. Suarez, C. Lagresle, A. M. Roche, J. Everett, A. Trinquand, M. Guisset, J. Xu Bayford, S. Hacein-Bey-Abina, A. Kauskot, R. Elfeky, C. Rivat, S. Abbas, H. B. Gaspar, E. Macintyre, C.
Published in: Nature Medicine, Issue 28, 2022, Page(s) 71-80, ISSN 1078-8956
Publisher: Nature Publishing Group
DOI: 10.1038/s41591-021-01641-x

Extensive multilineage analysis in patients with mixed chimerism after allogeneic transplantation for sickle cell disease: insight into hematopoiesis and engraftment thresholds for gene therapy

Author(s): Alessandra Magnani, Corinne Pondarré, Naïm Bouazza, Jeremy Magalon, Annarita Miccio, Emmanuelle Six, Cecile Roudaut, Cécile Arnaud, Annie Kamdem, Fabien Touzot, Aurélie Gabrion, Elisa Magrin, Chloé Couzin, Mathieu Fusaro, Isabelle André, Jean-Paul Vernant, Eliane Gluckman, Françoise Bernaudin, Dominique Bories, Marina Cavazzana
Published in: Haematologica, Issue 105, 2020, Page(s) 1240-1247, ISSN 0390-6078
Publisher: Ferrata Storti Foundation
DOI: 10.3324/haematol.2019.227561

Clonal tracking in gene therapy patients reveals a diversity of human hematopoietic differentiation programs

Author(s): Emmanuelle Six, Agathe Guilloux, Adeline Denis, Arnaud Lecoules, Alessandra Magnani, Romain Vilette, Frances Male, Nicolas Cagnard, Marianne Delville, Elisa Magrin, Laure Caccavelli, Cécile Roudaut, Clemence Plantier, Steicy Sobrino, John Gregg, Christopher L. Nobles, John K. Everett, Salima Hacein-Bey-Abina, Anne Galy, Alain Fischer, Adrian J. Thrasher, Isabelle André, Marina Cavazzana, Frederi
Published in: Blood, Issue 135, 2020, Page(s) 1219-1231, ISSN 0006-4971
Publisher: American Society of Hematology
DOI: 10.1182/blood.2019002350

Gene Therapy for β-Hemoglobinopathies

Author(s): Marina Cavazzana, Chiara Antoniani, Annarita Miccio
Published in: Molecular Therapy, Issue 25/5, 2017, Page(s) 1142-1154, ISSN 1525-0016
Publisher: Nature Publishing Group
DOI: 10.1016/j.ymthe.2017.03.024

Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus

Author(s): Chiara Antoniani, Vasco Meneghini, Annalisa Lattanzi, Tristan Felix, Oriana Romano, Elisa Magrin, Leslie Weber, Giulia Pavani, Sara El Hoss, Ryo Kurita, Yukio Nakamura, Thomas J. Cradick, Ante S. Lundberg, Matthew Porteus, Mario Amendola, Wassim El Nemer, Marina Cavazzana, Fulvio Mavilio, Annarita Miccio
Published in: Blood, Issue 131/17, 2018, Page(s) 1960-1973, ISSN 0006-4971
Publisher: American Society of Hematology
DOI: 10.1182/blood-2017-10-811505

An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype

Author(s): Leslie Weber, Valentina Poletti, Elisa Magrin, Chiara Antoniani, Samia Martin, Charles Bayard, Hanem Sadek, Tristan Felix, Vasco Meneghini, Michael N. Antoniou, Wassim El-Nemer, Fulvio Mavilio, Marina Cavazzana, Isabelle Andre-Schmutz, Annarita Miccio
Published in: Molecular Therapy - Methods & Clinical Development, Issue 10, 2018, Page(s) 268-280, ISSN 2329-0501
Publisher: Science Direct
DOI: 10.1016/j.omtm.2018.07.012

Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype

Author(s): Leslie Weber, Giacomo Frati, Tristan Felix, Giulia Hardouin, Antonio Casini, Clara Wollenschlaeger, Vasco Meneghini, Cecile Masson, Anne De Cian, Anne Chalumeau, Fulvio Mavilio, Mario Amendola, Isabelle Andre-Schmutz, Anna Cereseto, Wassim El Nemer, Jean-Paul Concordet, Carine Giovannangeli, Marina Cavazzana, Annarita Miccio
Published in: Science Advances, Issue 6, 2024, ISSN 2375-2548
Publisher: American Association for the Advancement of Science (AAAS)
DOI: 10.1126/sciadv.aay9392

Single-cell analysis of FOXP3 deficiencies in humans and mice unmasks intrinsic and extrinsic CD4+ T cell perturbations

Author(s): David Zemmour, Louis-Marie Charbonnier, Juliette Leon, Emmanuelle Six, Sevgi Keles, Marianne Delville, Mehdi Benamar, Safa Baris, Julien Zuber, Karin Chen, Benedicte Neven, Maria I. Garcia-Lloret, Frank M. Ruemmele, Carlo Brugnara, Nadine Cerf-Bensussan, Frederic Rieux-Laucat, Marina Cavazzana, Isabelle André, Talal A. Chatila, Diane Mathis, Christophe Benoist
Published in: Nature Immunology, Issue 22, 2023, Page(s) 607-619, ISSN 1529-2908
Publisher: Nature Publishing Group
DOI: 10.1038/s41590-021-00910-8