Associated Papers
CRISPR-Cas9n-mediated ELANE promoter editing for gene therapy of severe congenital neutropenia.
Nasri M et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy, 2024
Precision medicine: The use of tailored therapy in primary immunodeficiencies.
Pinto MV et al.
Frontiers in Immunology, 2022
CRISPR-Cas9-Mediated ELANE Mutation Correction in Hematopoietic Stem and Progenitor Cells to Treat Severe Congenital Neutropenia.
Tran NT et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy, 2020
The diversity of mutations and clinical outcomes for ELANE-associated neutropenia.
Makaryan V et al.
Current Opinion in Hematology, 2015
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