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Article

Comparison of Gene-Editing Approaches for Severe Congenital Neutropenia-Causing Mutations in the ELANE Gene.

The CRISPR journal - 1 Oct 2024

Ritter Malte Ulrich, Nasri Masoud, Dannenmann Benjamin, Mir Perihan, Secker Benjamin, Amend Diana, Klimiankou Maksim, Welte Karl, Skokowa Julia

Abstract excerpt

Safety considerations for gene therapies of inherited preleukemia syndromes, including severe congenital neutropenia (CN), are paramount. We compared several strategies for CRISPR/Cas9 gene editing of autosomal-dominant ELANE mutations in CD34+ cells from two CN patients head-to-head. We tested universal and allele-specific ELANE knockout, ELANE mutation correction by homology-directed repair (HDR) with AAV6, and...

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Share to Congenital Bone Marrow Failure Syndromes
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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

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Identifiers

PMID: 39436283
DOI: 10.1089/crispr.2024.0006
PMCID: PMC12344121

Genes

ELANE
CD34

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026.

Related research

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Research trail→

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Comparison of Gene-Editing Approaches for Severe Congenital Neutropenia-Causing Mutations in the ELANE Gene.PMID 39436283Read ↗

CRISPR-Cas9n-mediated ELANE promoter editing for gene therapy of severe congenital neutropenia.Semantic proximity Read ↗

CRISPR-Cas9-Mediated ELANE Mutation Correction in Hematopoietic Stem and Progenitor Cells to Treat Severe Congenital Neutropenia.Semantic proximity Read ↗

Dissecting ELANE neutropenia pathogenicity by human HSC gene editing.Semantic proximity Read ↗

A patient with severe congenital neutropenia harbors a missense ELANE mutation due to paternal germline mosaicism.Semantic proximity Read ↗

Paternal germline mosaicism of a missense mutation in ELANE in a Chinese male with severe congenital neutropeniaSemantic proximity Read ↗

Pathogenesis of ELANE-mutant severe neutropenia revealed by induced pluripotent stem cellsSemantic proximity Read ↗

Screening of genetic variants in ELANE mutation negative congenital neutropenia by next generation sequencing.Semantic proximity Read ↗

Understanding genetic heterogeneity in gene-edited hematopoietic stem cell products.Semantic proximity Read ↗

[Clinical and genetic analysis of two patients with congenital neutropenia caused by ELANE gene mutation].Semantic proximity Read ↗

Generation and characterization of a human iPSC line SANi007-A from a patient with a heterozygous dominant mutation in ELANE.Semantic proximity Read ↗

CRISPR/Cas9-mediated ELANE knockout enables neutrophilic maturation of primary hematopoietic stem and progenitor cells and induced pluripotent stem cells of severe congenital neutropenia patientsSemantic proximity Read ↗

Gene editing without <i>ex vivo</i> culture evades genotoxicity in human hematopoietic stem cellsSemantic proximity Read ↗

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