Exploiting ex vivo expansion and deep multiomics profiling to bring novel, efficient and safer hematopoietic stem cell gene therapies to clinical application

Project Information

X-PAND

Grant agreement ID: 101070950

DOI

10.3030/101070950

EC signature date 15 July 2022

Start date 1 October 2022

End date 30 September 2026

Funded under

• The European Innovation Council (EIC)

Total cost

€ 3 797 562,50

EU contribution

€ 3 797 562,50

Coordinated by

OSPEDALE SAN RAFFAELE SRL
Italy

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

Websites, patent fillings, videos etc. (1)

Setup of Project website, project logo and social media channels

Development and operation of the projects website development of project logo and development of social media channels

Publications

Peer reviewed articles (9)

p38 MAPK fuels proliferation stress and DNA damage impairing the functionality of genetically engineered hematopoietic stem and progenitor cells

Author(s): Raffaella Di Micco; Lucrezia della Volpe; Roberta Vacca; Federico Midena; Teresa Tavella; Matteo Maria Naldini; Matteo Barcella; Kety Giannetti; Samuele Ferrari; Stefano Beretta; Giacomo Farina; Laura Alessandrini; Antonella Santoro; Edoardo Carsana; Angelica Varesi; Diego Gilioli; Anastasia Conti; Ivan Merelli; Bernhard Gentner; Luigi Naldini
Published in: Cell Reports Medicine, Issue 5, 2024, ISSN 2666-3791
Publisher: Elsevier Inc.
DOI: 10.1016/J.XCRM.2024.101823

Protocol for optimizing culture conditions for ex vivo activation during CRISPR-Cas9 gene editing in human hematopoietic stem and progenitor cells

Author(s): Lucrezia della Volpe, Roberta Vacca, Raffaella Di Micco
Published in: STAR Protocols, Issue 6, 2025, ISSN 2666-1667
Publisher: Elsevier BV
DOI: 10.1016/J.XPRO.2025.103722

Beyond precision: evaluation of off-target clustered regularly interspaced short palindromic repeats/Cas9–mediated genome editing

Author(s): Irene Peña-Gutiérrez, Beatriz Olalla-Sastre, Paula Río, Juan R. Rodríguez-Madoz
Published in: Cytotherapy, Issue 27, 2025, ISSN 1465-3249
Publisher: Elsevier BV
DOI: 10.1016/J.JCYT.2024.10.010

CRISPR/Cas9-mediated gene editing. A promising strategy in hematological disorders

Author(s): Laura Ugalde, Sara Fañanas, Raúl Torres, Oscar Quintana-Bustamante, Paula Río
Published in: Cytotherapy, Issue 25, 2023, ISSN 1465-3249
Publisher: Elsevier BV
DOI: 10.1016/J.JCYT.2022.11.014

Genotoxic effects of base and prime editing in human hematopoietic stem cells

Author(s): Fiumara M, Ferrari S, Omer-Javed A, Beretta S, Albano L, Canarutto D, Varesi A, Gaddoni C, Brombin C, Cugnata F, Zonari E, Naldini MM, Barcella M, Gentner B, Merelli I, Naldini L.
Published in: Nature Biotechnology, 2023, ISSN 1546-1696
Publisher: Nature Biotechnology
DOI: 10.1038/s41587-023-01915-4

CRISPR-Cas9n-mediated ELANE promoter editing for gene therapy of severe congenital neutropenia

Author(s): Masoud Nasri, Malte U. Ritter, Perihan Mir, Benjamin Dannenmann, Masako M. Kaufmann, Patricia Arreba-Tutusaus, Yun Xu, Natalia Borbaran-Bravo, Maksim Klimiankou, Claudia Lengerke, Cornelia Zeidler, Toni Cathomen, Karl Welte, Julia Skokowa
Published in: Molecular Therapy, Issue 32, 2025, ISSN 1525-0016
Publisher: Elsevier BV
DOI: 10.1016/J.YMTHE.2024.03.037

Gene editing of NCF1 loci is associated with homologous recombination and chromosomal rearrangements

Author(s): Raimondi, Federica; Siow, Kah Mun; Wrona, Dominik; Fuster-García, Carla; Pastukhov, Oleksandr; Schmitz, Michael; Bargsten, Katja; Kissling, Lucas; Swarts, Daan C; Andrieux, Geoffroy; Cathomen, Toni; Modlich, Ute; Jinek, Martin; Siler, Ulrich; Reichenbach, Janine
Published in: Communications Biology, 2024, ISSN 2399-3642
Publisher: Nature Portfolio
DOI: 10.5167/UZH-263302

DNA-dependent protein kinase inhibitors PI-103 and samotolisib augment CRISPR/Cas9 knock-in efficiency in human T cells

Author(s): Emina Džafo, Morteza Hafezi, Greta Maria Paola Giordano Attianese, Patrick Reichenbach, Stephane Grillet, Hélène Garcia, Elisabetta Cribioli, Christel Voize, Stephanie Tissot, Romain Vuillefroy de Silly, George Coukos, Kirsten Scholten, Melita Irving, Bernhard Gentner
Published in: Cytotherapy, 2025, ISSN 1465-3249
Publisher: Elsevier BV
DOI: 10.1016/J.JCYT.2025.02.001

Donor insertion into <i>CX3CR1</i> allows epigenetic modulation of a constitutive promoter on hematopoietic stem cells and its activation upon myeloid differentiation

Author(s): Iris Ramos-Hernández; Carla Fuster-García; Araceli Aguilar-González; María L Lozano-Vinagre; Guillermo Guenechea-Amurrio; Francisco J Sanchez-Luque; Manuel A F V Gonçalves; Toni Cathomen; Pilar Muñoz; Francisco J Molina-Estévez; Francisco Martín
Published in: Nucleic Acids Research, 2025, ISSN 1362-4962
Publisher: Oxford University Press
DOI: 10.1093/NAR/GKAF344