Evidence map›Paper›PMID 40858805›Full record

ArticleLeukemia2025

SF3B1-mutant models of RNA mis-splicing uncover UBA1 as a therapeutic target in myelodysplastic neoplasms.

Jonas Thier, Sophia Hofmann, Katharina M Kirchhof, Gabriele Todisco, Teresa Mortera-Blanco, Ingrid Lilienthal, Dimitris C Kanellis, Indira Barbosa, Ann-Charlotte Björklund, André G Deslauriers and 7 more

Abstract read
In one paragraph

Article in Leukemia, 2025. 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. Splicing-driven post-translational dysregulation: a new frontier for precision cancer medicine and immunotherapy.Clinical & translational oncology : official publication of the Federation of Spanish Oncology Societies and of the National Cancer Institute of Mexico · 2026
    Review
  2. Article
  3. 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

17 authors.

Jonas ThierCenter for Hematology and Regenerative Medicine, Department of Medicine Huddinge, Karolinska Institutet, Huddinge, Sweden.ORCID 0009-0007-5226-0372
Sophia HofmannCenter for Hematology and Regenerative Medicine, Department of Medicine Huddinge, Karolinska Institutet, Huddinge, Sweden.ORCID 0009-0006-6625-8348
Katharina M KirchhofCenter for Hematology and Regenerative Medicine, Department of Medicine Huddinge, Karolinska Institutet, Huddinge, Sweden.ORCID 0000-0001-8412-4585
Gabriele TodiscoCenter for Hematology and Regenerative Medicine, Department of Medicine Huddinge, Karolinska Institutet, Huddinge, Sweden.ORCID 0000-0001-6583-3829
Teresa Mortera-BlancoCenter for Hematology and Regenerative Medicine, Department of Medicine Huddinge, Karolinska Institutet, Huddinge, Sweden.ORCID 0000-0002-8399-1430
Ingrid LilienthalDivision of Pediatric Oncology and Surgery, Department of Women's and Children's Health, Karolinska Institutet, Stockholm, Sweden.ORCID 0000-0001-5270-7268
Dimitris C KanellisDepartment of Medical Biochemistry and Biophysics, Science for Life Laboratory, Division of Genome Biology, Karolinska Institutet, Stockholm, Sweden.ORCID 0000-0001-8690-2010
Indira BarbosaCenter for Hematology and Regenerative Medicine, Department of Medicine Huddinge, Karolinska Institutet, Huddinge, Sweden.ORCID 0009-0004-1171-2932
Ann-Charlotte BjörklundCenter for Hematology and Regenerative Medicine, Department of Medicine Huddinge, Karolinska Institutet, Huddinge, Sweden.ORCID 0009-0008-3745-503X
André G DeslauriersGlobal Drug Discovery, Novo Nordisk A/S, Måløv, Denmark.ORCID 0000-0002-2854-5681
Jiri BartekDepartment of Medical Biochemistry and Biophysics, Science for Life Laboratory, Division of Genome Biology, Karolinska Institutet, Stockholm, Sweden.ORCID 0000-0003-2013-7525
Nikolas HeroldDivision of Pediatric Oncology and Surgery, Department of Women's and Children's Health, Karolinska Institutet, Stockholm, Sweden.ORCID 0000-0001-9468-4543
Elli PapaemmanuilComputational Oncology Service, Department of Epidemiology and Biostatistics, Memorial Sloan Kettering Cancer Center, New York, NY, USA.ORCID 0000-0003-1709-8983
Eirini P PapapetrouDepartment of Oncological Sciences, Icahn School of Medicine at Mount Sinai, New York, NY, USA.ORCID 0000-0001-7002-417X
Eva Hellström-LindbergCenter for Hematology and Regenerative Medicine, Department of Medicine Huddinge, Karolinska Institutet, Huddinge, Sweden.ORCID 0000-0002-7839-3743
Pedro L MouraCenter for Hematology and Regenerative Medicine, Department of Medicine Huddinge, Karolinska Institutet, Huddinge, Sweden.ORCID 0000-0002-0493-5394
Vanessa LundinCenter for Hematology and Regenerative Medicine, Department of Medicine Huddinge, Karolinska Institutet, Huddinge, Sweden. vanessa.lundin@ki.se.ORCID 0000-0003-2335-3370

Funding

X-RAY CRYSTALLOGRAPHYP30CA008748 · NCI · SLOAN-KETTERING INSTITUTE FOR CANCER RES · PI SELWYN M VICKERS · 1985 to 2026
$347.4M
Cancerfonden (Swedish Cancer Society) 19 0200Cancerfonden (Swedish Cancer Society) 21 0340Knut och Alice Wallenbergs Stiftelse (Knut and Alice Wallenberg Foundation) 2017.0359NCI NIH HHS P30 CA008748Vetenskapsrådet (Swedish Research Council) 2020-01902Vetenskapsrådet (Swedish Research Council) 211133
6 · The paper itself

Abstract

Myelodysplastic syndromes with somatic mutations in the splicing factor SF3B1 gene (MDS-SF3B1) result in RNA mis-splicing, erythroid dysplasia and ultimately refractory anemia. Precision medicine approaches for MDS-SF3B1 remain challenging due to both the complexity of the mis-splicing landscape and its evaluation in disease-accurate models. To uncover novel RNA mis-splicing events, isogenic SF3B1

Indexed as

MutationMyelodysplastic SyndromesPhosphoproteinsRNA SplicingRNA Splicing FactorsUbiquitin-Activating EnzymesHumansInduced Pluripotent Stem CellsPhosphoproteinsRNA Splicing FactorsSF3B1 protein, humanUBA1 protein, humanUbiquitin-Activating Enzymes

Identifiers

PMID40858805
PMCPMC12589102

What OpenQuestion holds

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Registered trials

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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.