Development of Innovative Therapeutic Strategies for beta-hemoglobinopathies

Project Information

DITSB

Grant agreement ID: 865797

DOI

10.3030/865797

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 (8)

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

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

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

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

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