p38 MAPK fuels proliferation stress and DNA damage impairing the functionality of genetically engineered hematopoietic stem and progenitor cells
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Autores:
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
Publicado en:
Cell Reports Medicine, Edición 5, 2024, ISSN 2666-3791
Editor:
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
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Autores:
Lucrezia della Volpe, Roberta Vacca, Raffaella Di Micco
Publicado en:
STAR Protocols, Edición 6, 2025, ISSN 2666-1667
Editor:
Elsevier BV
DOI:
10.1016/J.XPRO.2025.103722
Genotoxic effects of base and prime editing in human hematopoietic stem cells
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Autores:
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.
Publicado en:
Nature Biotechnology, 2023, ISSN 1546-1696
Editor:
Nature Biotechnology
DOI:
10.1038/s41587-023-01915-4
CRISPR-Cas9n-mediated ELANE promoter editing for gene therapy of severe congenital neutropenia
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Autores:
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
Publicado en:
Molecular Therapy, Edición 32, 2025, ISSN 1525-0016
Editor:
Elsevier BV
DOI:
10.1016/J.YMTHE.2024.03.037
Gene editing of NCF1 loci is associated with homologous recombination and chromosomal rearrangements
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Autores:
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
Publicado en:
Communications Biology, 2024, ISSN 2399-3642
Editor:
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
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Autores:
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
Publicado en:
Cytotherapy, 2025, ISSN 1465-3249
Editor:
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
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Autores:
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
Publicado en:
Nucleic Acids Research, 2025, ISSN 1362-4962
Editor:
Oxford University Press
DOI:
10.1093/NAR/GKAF344
