Evidence map›Paper›PMID 39436283›Full record

ArticleThe CRISPR journal2024

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

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

Abstract readComparative Study
In one paragraph

Article in The CRISPR journal, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

3 citing papers in PubMed.

  1. Review
  2. Review
  3. Cyclic neutropenia: a case report and literature review.American journal of translational research · 2025
    Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

9 authors.

Malte Ulrich RitterDepartment of Hematology, Oncology, Immunology and Rheumatology, University Hospital Tübingen, Tübingen, Germany.
Masoud NasriDepartment of Hematology, Oncology, Immunology and Rheumatology, University Hospital Tübingen, Tübingen, Germany.
Benjamin DannenmannDepartment of Hematology, Oncology, Immunology and Rheumatology, University Hospital Tübingen, Tübingen, Germany.
Perihan MirDepartment of Hematology, Oncology, Immunology and Rheumatology, University Hospital Tübingen, Tübingen, Germany.
Benjamin SeckerDepartment of Hematology, Oncology, Immunology and Rheumatology, University Hospital Tübingen, Tübingen, Germany.
Diana AmendDepartment of Hematology, Oncology, Immunology and Rheumatology, University Hospital Tübingen, Tübingen, Germany.
Maksim KlimiankouDepartment of Hematology, Oncology, Immunology and Rheumatology, University Hospital Tübingen, Tübingen, Germany.
Karl WelteDepartment of Pediatric Hematology, Oncology and Bone Marrow Transplantation, University Hospital Tübingen, Tübingen, Germany.
Julia SkokowaDepartment of Hematology, Oncology, Immunology and Rheumatology, University Hospital Tübingen, Tübingen, Germany.ORCID 0000-0002-4399-2324

Funding

Systems Biology of Bone Marrow Failure and MDS for Precision MedicineRC2DK122533 · NIDDK · BOSTON CHILDREN'S HOSPITAL · PI FIGUEROA, MARIA EUGENIA, FLEMING, MARK D · 2019 to 2023
$6.4M
Severe Chronic Neutropenia International RegistryR24AI162637 · NIAID · BOSTON CHILDREN'S HOSPITAL · PI PETER E NEWBURGER, Akiko Shimamura · 2022 to 2026
$5.8M
NIAID NIH HHS R24 AI162637NIDDK NIH HHS RC2 DK122533
6 · The paper itself

Abstract

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

Indexed as

Congenital Bone Marrow Failure SyndromesCRISPR-Cas SystemsGene EditingGenetic TherapyMutationNeutropeniaAllelesHumansInduced Pluripotent Stem CellsLeukocyte ElastaseRecombinational DNA RepairELANE protein, humanLeukocyte Elastase

Identifiers

PMID39436283
PMCPMC12344121

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.