Development of Innovative Therapeutic Strategies for beta-hemoglobinopathies

Project Information

DITSB

Grant agreement ID: 865797

DOI

10.3030/865797

Project closed

EC signature date 24 March 2020

Start date 1 June 2020

End date 31 December 2025

Funded under

• EXCELLENT SCIENCE - European Research Council (ERC)

Total cost

€ 2 000 000,00

EU contribution

€ 2 000 000,00

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 (14)

Une approche CRISPR/Cas pour traiter les β-hémoglobinopathies

Author(s): Megane Brusson, Annarita Miccio
Published in: médecine/sciences, Issue 41, 2025, Page(s) 33-39, ISSN 0767-0974
Publisher: Editions E D K
DOI: 10.1051/medsci/2024191

CRISPRthripsis: The Risk of CRISPR/Cas9-induced Chromothripsis in Gene Therapy

Author(s): Mario Amendola; Mégane Brusson; Annarita Miccio
Published in: Stem Cells Transl Med, Issue 1, 2022, ISSN 2157-6564
Publisher: AlphaMed Press
DOI: 10.1093/stcltm/szac064

Base editing of β ⁰ -thalassemia mutations as a therapeutic strategy for severe β-hemoglobinopathies

Author(s): Giulia Hardouin, Pierre Martinucci, Samantha Scaramuzza, Panagiotis Antoniou, Federico Corradi, Alexandra Tachtsidi, Guillaume Corre, Margaux Mombled, Jessika Chermont, Sandra Manceau, Cécile Rouillon, Cécile Masson, Laure Joseph, Isabelle Thuret, Catherine Badens, Sarah Szepetowsky, Eric Allemand, Mario Amendola, Oriana Romano, Giuliana Ferrari, Annarita Miccio
Published in: Science Translational Medicine, Issue 17, 2025, ISSN 1946-6234
Publisher: American Association for the Advancement of Science
DOI: 10.1126/scitranslmed.adt8617

Adenine base editor-mediated correction of the common and severe IVS1-110 (G>A) β-thalassemia mutation

Author(s): Hardouin G, Antoniou P, Martinucci P, Felix T, Manceau S, Joseph L, Masson C, Scaramuzza S, Ferrari G, Cavazzana M, Miccio A
Published in: Blood, Issue continous, 2022, ISSN 0006-4971
Publisher: American Society of Hematology
DOI: 10.1182/blood.2022016629

Novel genome-editing-based approaches to treat motor neuron diseases: Promises and challenges.

Author(s): Miccio A*, Antoniou P, Ciura S, Kabashi E.*
Published in: Mol Ther., Issue continous, 2021, ISSN 1525-0016
Publisher: Nature Publishing Group
DOI: 10.1016/j.ymthe.2021.04.003

Advanced delivery systems for gene editing: A comprehensive review from the GenE-HumDi COST Action Working Group

Author(s): Alessia Cavazza, Francisco J. Molina-Estévez, Álvaro Plaza Reyes, Victor Ronco, Asma Naseem, Špela Malenšek, Peter Pečan, Annalisa Santini, Paula Heredia, Araceli Aguilar-González, Houria Boulaiz, Qianqian Ni, Marina Cortijo-Gutierrez, Kristina Pavlovic, Inmaculada Herrera, Berta de la Cerda, Emilio M. Garcia-Tenorio, Eva Richard, Sergio Granados-Principal, Arístides López-Márquez, Marian
Published in: Molecular Therapy Nucleic Acids, Issue 36, 2025, Page(s) 102457, ISSN 2162-2531
Publisher: Nature Publishing Group
DOI: 10.1016/j.omtn.2025.102457

Epigenetic Regulation of β-Globin Genes and the Potential to Treat Hemoglobinopathies through Epigenome Editing

Author(s): Letizia Fontana, Zoe Alahouzou, Annarita Miccio, Panagiotis Antoniou
Published in: Genes, Issue 14, 2025, Page(s) 577, ISSN 2073-4425
Publisher: Multidisciplinary Digital Publishing Institute (MDPI)
DOI: 10.3390/genes14030577

Recent progress in genome editing for gene therapy applications: the French perspective

Author(s): Amendola M, Bedel A, Buj Bello A, Carrara M, Concordet JP, Frati G, Gilot D, Giovannangeli C, Guttierrez A, Laurent M, Miccio A, Moreau-Gaudry F, Sourd C, Valton J, Verhoeyen E.
Published in: Hum Gene Ther., Issue continous, 2021, ISSN 1043-0342
Publisher: Mary Ann Liebert Inc.
DOI: 10.1089/hum.2021.191

Genome Editing for β-Hemoglobinopathies: Advances and Challenges.

Author(s): Giacomo Frati; Annarita Miccio
Published in: Journal of Clinical Medicine, Issue 1, 2021, ISSN 2077-0383
Publisher: Multidisciplinary Digital Publishing Institute (MDPI)
DOI: 10.3390/jcm10030482

Analysis of Globin Chains from Human Erythroid Cells.

Author(s): Chalumeau A, Frati G, Magrin E, Miccio A
Published in: Bio Protoc., Issue continous, 2021, ISSN 2331-8325
Publisher: Bio Protoc.
DOI: 10.21769/bioprotoc.3899

Multiplex base editing of BCL11A regulatory elements to treat sickle cell disease

Author(s): Letizia Fontana, Pierre Martinucci, Simone Amistadi, Tristan Felix, Margaux Mombled, Alexandra Tachtsidi, Guillaume Corre, Anne Chalumeau, Giulia Hardouin, Jeanne Martin, Oriana Romano, Mario Amendola, Panagiotis Antoniou, Annarita Miccio
Published in: Cell Reports Medicine, Issue 6, 2025, Page(s) 102376, ISSN 2666-3791
Publisher: Elsevier BV
DOI: 10.1016/j.xcrm.2025.102376

Base editing strategies to hematological genetic disorders.

Author(s): Antoniou P, Miccio A * Brusson M *.
Published in: Front Genome Ed., Issue continous, 2021, ISSN 2673-3439
Publisher: Frontiers
DOI: 10.3389/fgeed.2021.618406

Safety and efficacy studies of CRISPR-Cas9 treatment of sickle cell disease highlights disease-specific responses

Author(s): Giacomo Frati, Megane Brusson, Gilles Sartre, Bochra Mlayah, Tristan Felix, Anne Chalumeau, Panagiotis Antoniou, Giulia Hardouin, Jean-Paul Concordet, Oriana Romano, Giandomenico Turchiano, Annarita Miccio
Published in: Molecular Therapy, Issue 32, 2025, Page(s) 4337-4352, ISSN 1525-0016
Publisher: Nature Publishing Group
DOI: 10.1016/j.ymthe.2024.07.015

Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression

Author(s): Antoniou P, Hardouin G, Martinucci P, Frati G, Felix T, Chalumeau A, Fontana L, Martin J, Masson C, Brusson M, Maule G, Rosello M, Giovannangeli C, Abramowski V, de Villartay JP, Concordet JP, Del Bene F, El Nemer W, Amendola M, Cavazzana M, Cereseto A, Romano O, Miccio A.
Published in: Nat Commun., Issue continous, 2022, ISSN 2041-1723
Publisher: Nature Publishing Group
DOI: 10.1038/s41467-022-34493-1