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CORDIS

Assessing efficacy and safety of genome EDITing approaches for Sickle Cell Disease

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 .

Deliverables

Data Management Plan 3 (opens in new window)

We will revise the data management plan (see paragraph 1.2.6)and with the final release expected at month 36.

Data Management Plan 2 (opens in new window)

We will revise the data management plan (see paragraph 1.2.6) at month 18

Data Management Plan 1 (opens in new window)

A document describing the data management plan (see paragraph 1.2.6) will be produced and provided at month 6

Project website and logo (opens in new window)

A web page will be created in English for information of patients, families, patient organizations, the general public, health care providers, financial actors on the aims and progress of the project.

Dissemination and communication plan 2 (opens in new window)

The dissemination and communication plan will be updated during the project by selecting and prioritizing the most timely and relevant dissemination and communication activities.

Publications

Clinical genome editing to treat sickle cell disease—A brief update (opens in new window)

Author(s): Parinaz Zarghamian; Julia Klermund; Toni Cathomen
Published in: Crossref, 2023, ISSN 2296-858X
Publisher: Frontiers in Medecine
DOI: 10.3389/fmed.2022.1065377

Nature Communications (opens in new window)

Author(s): Louis C. Dacquay; Panagiotis Antoniou; Astrid Mentani; Niklas Selfjord; Hanna Mårtensson; Pei-Pei Hsieh; Salman Mustfa; George Thom; Sandra Wimberger; Mike Firth; Nina Akrap; Marcello Maresca; Martin Peterka
Published in: Nature Communications, 2025, ISSN 2041-1723
Publisher: Nature Publishing Group
DOI: 10.1038/S41467-025-59708-Z

Molecular Therapy - Nucleic Acids (opens in new window)

Author(s): Venkatesan et al.
Published in: Molecular therapy Nucleic acids, 2023, ISSN 2162-2531
Publisher: Nature Publishing Group
DOI: 10.1016/j.omtn.2023.04.024

Nature Communications (opens in new window)

Author(s): Grégoire Cullot; Eric J. Aird; Moritz F. Schlapansky; Charles D. Yeh; Lilly van de Venn; Iryna Vykhlyantseva; Susanne Kreutzer; Dominic Mailänder; Bohdan Lewków; Julia Klermund; Christian Montellese; Martina Biserni; Florian Aeschimann; Cédric Vonarburg; Helmuth Gehart; Toni Cathomen; Jacob E. Corn
Published in: Nature Biotechnology, 2024, ISSN 2041-1723
Publisher: Nature Publishing Group
DOI: 10.3929/ETHZ-B-000709646

Molecular Therapy - Nucleic Acids (opens in new window)

Author(s): Nechama Kalter; Carla Fuster-García; Alfredo Silva; Víctor Ronco-Díaz; Stefano Roncelli; Giandomenico Turchiano; Jan Gorodkin; Toni Cathomen; Karim Benabdellah; Ciaran Lee; Ayal Hendel
Published in: Molecular Therapy Nucleic Acids, 2025, ISSN 2162-2531
Publisher: Cell Press
DOI: 10.1016/J.OMTN.2025.102636

Molecular Therapy - Nucleic Acids (opens in new window)

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, 2024, ISSN 2162-2531
Publisher: Nature Publishing Group
DOI: 10.1101/2024.01.14.575586

Nature Communications (opens in new window)

Author(s): Steicy Sobrino; Laure Joseph; Elisa Magrin; Anne Chalumeau; Nicolas Hebert; Alice Corsia; Adeline Denis; Cécile Roudaut; Clotilde Aussel; Olivia Leblanc; Mégane Brusson; Tristan Felix; Jean-Sebastien Diana; Angelina Petrichenko; Jana El Etri; Auria Godard; Eden Tibi; Sandra Manceau; Jean Marc Treluyer; Fulvio Mavilio; Frederic D. Bushman; Ambroise Marcais; Martin Castelle; Benedicte Neven; Olivier Hermine; Sylvain Renolleau; Alessandra Magnani; Vahid Asnafi; Wassim El Nemer; Pablo Bartolucci; Emmanuelle Six; Michaela Semeraro; Annarita Miccio; Marina Cavazzana
Published in: Nature Communications, 2025, ISSN 2041-1723
Publisher: Nature Publishing Group
DOI: 10.1038/S41467-025-58321-4

Une approche CRISPR/Cas pour traiter les β-hémoglobinopathies (opens in new window)

Author(s): Megane Brusson; Annarita Miccio
Published in: médecine/sciences, 2025, ISSN 2076-3271
Publisher: Multidisciplinary Digital Publishing Institute
DOI: 10.1051/MEDSCI/2024191

Molecular Therapy - Nucleic Acids (opens in new window)

Author(s): Frati, Giacomo; Brusson, Megane; Sartre, Gilles; Mlayah, Bochra; Felix, Tristan; Chalumeau, Anne; Antoniou, Panagiotis; Hardouin, Giulia; Concordet, Jean-Paul; Romano, Oriana; Turchiano, Giandomenico; Miccio, Annarita
Published in: Molecular Therapy, 2024, ISSN 2162-2531
Publisher: Cell Press
DOI: 10.1016/J.YMTHE.2024.07.015

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