Evidence map›Paper›PMID 41171815›Full record

ArticlePloS one2025

A porcine model of Fanconi anemia.

Brandon Hergert, Kristin M Whitworth, Devorah C Goldman, Lisa Moreau, Kelsey McQueen, Kalindi Parmar, Alan D'Andrea, Melissa S Samuel, Kevin D Wells, Randall S Prather and 3 more

Abstract read
In one paragraph

Article in PloS one, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

13 authors.

Brandon HergertDepartment of Pediatrics, Papé Family Pediatric Research Institute, OHSU, Portland, Oregon.
Kristin M WhitworthDivision of Animal Sciences, University of Missouri, National Swine Resource and Research Center, Columbia, Missouri, United State of America.
Devorah C GoldmanDepartment of Pediatrics, Papé Family Pediatric Research Institute, OHSU, Portland, Oregon.
Lisa MoreauDana Farber Cancer Institute, Harvard University, Boston, Massachusetts, United State of America.
Kelsey McQueenDana Farber Cancer Institute, Harvard University, Boston, Massachusetts, United State of America.
Kalindi ParmarDana Farber Cancer Institute, Harvard University, Boston, Massachusetts, United State of America.
Alan D'AndreaDana Farber Cancer Institute, Harvard University, Boston, Massachusetts, United State of America.
Melissa S SamuelDivision of Animal Sciences, University of Missouri, National Swine Resource and Research Center, Columbia, Missouri, United State of America.
Kevin D WellsDivision of Animal Sciences, University of Missouri, National Swine Resource and Research Center, Columbia, Missouri, United State of America.
Randall S PratherDivision of Animal Sciences, University of Missouri, National Swine Resource and Research Center, Columbia, Missouri, United State of America.
Craig DorellDepartment of Pediatrics, Papé Family Pediatric Research Institute, OHSU, Portland, Oregon.
Markus GrompeDepartment of Pediatrics, Papé Family Pediatric Research Institute, OHSU, Portland, Oregon.
William H FlemingDepartment of Pediatrics, Papé Family Pediatric Research Institute, OHSU, Portland, Oregon.

Funding

Resource Core for National Swine Resource and Research Center (NSRRC) Years 21-25U42OD011140 · OD · UNIVERSITY OF MISSOURI-COLUMBIA · PI Kiho Lee · 2012 to 2026
$26.7M
NIH HHS U42 OD011140
6 · The paper itself

Abstract

Although small animal models of Fanconi anemia (FA) are useful, they do not faithfully replicate many of the clinical features seen in FA patients. We reasoned that a porcine model of FA with its similar physiology and a relatively long lifespan would produce a phenotype more similar to human FA. Targeting FANCA in domestic swine resulted in skeletal abnormalities and extreme sensitivity to interstrand DNA cross-linking agents. In addition, FANCA disruption followed by mitomycin C treatment resulted in a > 10-fold increase in chromosomal radials, a finding that is considered diagnostic for human FA. Bone marrow derived, hematopoietic progenitor cells from a FANCA null pig showed a 75% reduction in colony forming activity compared to wild type. Evaluation of steady state hematopoiesis in the peripheral blood revealed the gradual development of red cell macrocytosis and a reduction in circulating neutrophils. Targeting of FANCD2 failed to produce any biallelic animals demonstrating the loss of FANCD2 function is embryonic lethal in pigs. These results indicate that a porcine model of FANCA holds promise for the development of strategies to prevent the development of bone marrow failure and malignancies in patients with FA.

Indexed as

Disease Models, AnimalFanconi AnemiaAnimalsFanconi Anemia Complementation Group A ProteinFanconi Anemia Complementation Group D2 ProteinHematopoiesisHematopoietic Stem CellsHumansMitomycinSwineFanconi Anemia Complementation Group A ProteinFanconi Anemia Complementation Group D2 ProteinMitomycin

Identifiers

PMID41171815
PMCPMC12578174

What OpenQuestion holds

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LicenceCC BY
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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.