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

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Authors: 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

Journal title: Molecular Therapy

Journal number: 32

Journal publisher: Elsevier BV

Published year: 2025

DOI identifier: 10.1016/J.YMTHE.2024.03.037

ISSN: 1525-0016