CRISPR-Cas9n-mediated ELANE promoter editing for gene therapy of severe congenital neutropenia
Masoud Nasri, Malte Ritter, Perihan Mir, Benjamin Dannenmann, Masako Monika Kaufmann, Patricia Arreba-Tutusaus, Yun Xu, Natalia Borbaran-Bravo, Maksim Klimiankou, Claudia Lengerke, Cornelia Zeidler, Toni Cathomen, Karl Welte, Julia Skokowa
Topics & Concepts
Congenital NeutropeniaCyclic neutropeniaNeutropeniaBiologyCancer researchGenetic enhancementImmunologyGeneticsGeneChemotherapyBlood disorders and treatmentsImmunodeficiency and Autoimmune DisordersCRISPR and Genetic Engineering