Evidence map›Paper›PMID 42201986›Full record

ArticlePrenatal diagnosis2026

Evaluation of Maternal Safety Following Prenatal Cell and Gene Therapy for Hemophilia A.

Quan Minh Pham, Martin Rodriguez, Ritu M Ramamurthy, Sunil George, Walaa M Mohamed, Oluwaseun Babatunde, Michael Gautreaux, Christopher B Doering, H Trent Spencer, Anthony Atala and 2 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Prenatal diagnosis, 2026. 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

12 authors.

Quan Minh PhamWake Forest Institute for Regenerative Medicine, Fetal Research and Therapy Program, Wake Forest University School of Medicine (WFUSM), Winston-Salem, North Carolina, USA.
Martin RodriguezWake Forest Institute for Regenerative Medicine, Fetal Research and Therapy Program, Wake Forest University School of Medicine (WFUSM), Winston-Salem, North Carolina, USA.
Ritu M RamamurthyWake Forest Institute for Regenerative Medicine, Fetal Research and Therapy Program, Wake Forest University School of Medicine (WFUSM), Winston-Salem, North Carolina, USA.
Sunil GeorgeWake Forest Institute for Regenerative Medicine, Fetal Research and Therapy Program, Wake Forest University School of Medicine (WFUSM), Winston-Salem, North Carolina, USA.
Walaa M MohamedWake Forest Institute for Regenerative Medicine, Fetal Research and Therapy Program, Wake Forest University School of Medicine (WFUSM), Winston-Salem, North Carolina, USA.
Oluwaseun BabatundeWake Forest Institute for Regenerative Medicine, Fetal Research and Therapy Program, Wake Forest University School of Medicine (WFUSM), Winston-Salem, North Carolina, USA.
Michael GautreauxHLA/Immunogenetics and Immunodiagnostics Laboratories, Winston-Salem, North Carolina, USA.
Christopher B DoeringChildren's Healthcare of Atlanta and Dept. of Pediatrics, Aflac Cancer and Blood Disorders Center, Emory University, Atlanta, Georgia, USA.
H Trent SpencerChildren's Healthcare of Atlanta and Dept. of Pediatrics, Aflac Cancer and Blood Disorders Center, Emory University, Atlanta, Georgia, USA.
Anthony AtalaWake Forest Institute for Regenerative Medicine, Fetal Research and Therapy Program, Wake Forest University School of Medicine (WFUSM), Winston-Salem, North Carolina, USA.
Christopher D PoradaWake Forest Institute for Regenerative Medicine, Fetal Research and Therapy Program, Wake Forest University School of Medicine (WFUSM), Winston-Salem, North Carolina, USA.ORCID https://orcid.org/0000-0002-7321-7556
Graça Almeida-PoradaWake Forest Institute for Regenerative Medicine, Fetal Research and Therapy Program, Wake Forest University School of Medicine (WFUSM), Winston-Salem, North Carolina, USA.

Funding

Prenatal Cell and Gene Therapy for Hemophilia AR01HL135853 · NHLBI · WAKE FOREST UNIVERSITY HEALTH SCIENCES · PI ALMEIDA-PORADA, GRACA DUARTE, PORADA, CHRISTOPHER D · 2017 to 2020
$2.8M
cGMP Manufacture Of FVIII-Expressing Placental Cells For Hemophilia AU01HL148681 · NHLBI · WAKE FOREST UNIVERSITY HEALTH SCIENCES · PI Graca Duarte Almeida-Porada · 2019 to 2026
$1.6M
HHMI Gilliam Graduate FellowshipsNHLBI NIH HHS R01 HL135853NHLBI NIH HHS U01 HL148681NIH HHS R01 HL135853NIH HHS U0 HL1148681
6 · The paper itself

Abstract

objectiveMaternal safety is extremely important in prenatal therapies, especially if the product could impact the pregnant women.

methodsThis study uses fetal sheep as a model to investigate whether intraperitoneal administration of human placental cells modified to encode a bioengineered fVIII transgene (mcoET3) exposes the ewes to the transplanted product (PLC-mcoET3) or its secreted proteins, thereby inducing an immune response.

resultsMixed-lymphocyte reactions and sensitive multiplex bead assays demonstrated that prenatal treatment did not immunize the ewes to the transplanted cells or induce anti-HLA Class I and Class II antibodies. ELISpot assays and mcoET3-specific ELISAs demonstrated the absence of mcoET3-specific T

conclusionOverall, these studies suggest that the administration of PLC-mcoET3 to the fetus during gestation does not result in detectable maternal exposure to the cells or gene products, attesting to the safety of this approach.

Identifiers

PMID42201986
PMCPMC13436575

What Socratic holds

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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 Socratic graph.