Evidence map›Paper›PMID 39653038›Full record

ArticleMolecular therapy : the journal of the American Society of Gene Therapy2025

Flavopiridol restores granulopoiesis in experimental models of severe congenital neutropenia.

Masoud Nasri, Benjamin Dannenmann, Larissa Doll, Betül Findik, Franka Bernhard, Sergey Kandabarau, Maksim Klimiankou, Meinrad Gawaz, Claudia Lengerke, Cornelia Zeidler and 2 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing 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

2 citing papers in PubMed.

  1. Review
  2. Review
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

12 authors.

Masoud NasriDepartment of Oncology, Hematology, Clinical Immunology and Rheumatology, University Hospital Tuebingen, 72076 Tuebingen, Germany.
Benjamin DannenmannDepartment of Oncology, Hematology, Clinical Immunology and Rheumatology, University Hospital Tuebingen, 72076 Tuebingen, Germany.
Larissa DollDepartment of Oncology, Hematology, Clinical Immunology and Rheumatology, University Hospital Tuebingen, 72076 Tuebingen, Germany.
Betül FindikDepartment of Oncology, Hematology, Clinical Immunology and Rheumatology, University Hospital Tuebingen, 72076 Tuebingen, Germany.
Franka BernhardDepartment of Oncology, Hematology, Clinical Immunology and Rheumatology, University Hospital Tuebingen, 72076 Tuebingen, Germany.
Sergey KandabarauDepartment of Oncology, Hematology, Clinical Immunology and Rheumatology, University Hospital Tuebingen, 72076 Tuebingen, Germany.
Maksim KlimiankouDepartment of Oncology, Hematology, Clinical Immunology and Rheumatology, University Hospital Tuebingen, 72076 Tuebingen, Germany.
Meinrad GawazDepartment of Cardiology and Angiology, University Hospital Tuebingen, 72076 Tuebingen, Germany.
Claudia LengerkeDepartment of Oncology, Hematology, Clinical Immunology and Rheumatology, University Hospital Tuebingen, 72076 Tuebingen, Germany.
Cornelia ZeidlerDepartment of Oncology, Hematology, Clinical Immunology and Rheumatology, University Hospital Tuebingen, 72076 Tuebingen, Germany.
Karl WelteDepartment of Oncology, Hematology, Clinical Immunology and Rheumatology, University Hospital Tuebingen, 72076 Tuebingen, Germany; Department of Pediatric Hematology, Oncology, and Bone Marrow Transplantation, Children's Hospital, University Hospital Tuebingen, 72076 Tuebingen, Germany.
Julia SkokowaDepartment of Oncology, Hematology, Clinical Immunology and Rheumatology, University Hospital Tuebingen, 72076 Tuebingen, Germany; Gene and RNA Therapy Center (GRTC), Tuebingen University, 72076 Tuebingen, Germany. Electronic address: julia.skokowa@med.uni-tuebingen.de.

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

Severe congenital neutropenia (CN) patients require life-long treatment with recombinant human granulocyte colony-stimulating factor (rhG-CSF), but some show no response. We sought to establish a therapy for CN that targets signaling pathways causing maturation arrest of granulocytic progenitors. We developed an isogenic induced pluripotent stem cell (iPSC) in vitro model of CN associated with ELANE mutations (ELANE-CN) and performed an in silico drug repurposing analysis of the transcriptomics of iPSC-generated hematopoietic stem and progenitor cells. We identified flavopiridol, a Food and Drug Administration (FDA)-approved pan-cyclin-dependent kinase inhibitor, as a potential therapeutic. Treatment with low-dose flavopiridol rescued defective granulopoiesis in primary CD34

Indexed as

Congenital Bone Marrow Failure SyndromesFlavonoidsGranulocytesNeutropeniaPiperidinesAnimalsCell DifferentiationDisease Models, AnimalHematopoietic Stem CellsHumansInduced Pluripotent Stem CellsMutationZebrafishalvocidibFlavonoidsPiperidinesCEBPAcyclin-dependent kinasesflavopiridolin silico drug repurposingisogenic patient-derived iPSC linesLINCS L1000severe congenital neutropeniazebrafish models of congenital neutropenia

Identifiers

PMID39653038
PMCPMC12172196

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.