Evidence map›Paper›PMID 40909633›Full record

ArticlebioRxiv : the preprint server for biology2025

Lentiviral-mediated gene complementation rescues pathogenic

Ashley L Cooney, Shakayla Lamer, Ping Yang, Daniel J Wegner, Frances V White, F Sessions Cole, Chris Wohlford-Lenane, Erin Hennessey, Pushpinder Bawa, Darrell N Kotton and 3 more

Abstract readPreprint
In one paragraph

Article in bioRxiv : the preprint server for biology, 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

5 · Who and what money

Authors and funding

13 authors.

Ashley L CooneyUniversity of Iowa, Stead Family Department of Pediatrics, Iowa City, IA, 52245, USA.ORCID 0000-0003-2717-9138
Shakayla LamerUniversity of Iowa, Stead Family Department of Pediatrics, Iowa City, IA, 52245, USA.
Ping YangEdward Mallinckrodt Department of Pediatrics, Washington University in St. Louis, St. Louis Children's Hospital, St. Louis, MO 63110, USA.
Daniel J WegnerEdward Mallinckrodt Department of Pediatrics, Washington University in St. Louis, St. Louis Children's Hospital, St. Louis, MO 63110, USA.
Frances V WhiteEdward Mallinckrodt Department of Pediatrics, Washington University in St. Louis, St. Louis Children's Hospital, St. Louis, MO 63110, USA.
F Sessions ColeEdward Mallinckrodt Department of Pediatrics, Washington University in St. Louis, St. Louis Children's Hospital, St. Louis, MO 63110, USA.ORCID 0000-0002-3797-9369
Chris Wohlford-LenaneUniversity of Iowa, Stead Family Department of Pediatrics, Iowa City, IA, 52245, USA.
Erin HennesseyCenter for Regenerative Medicine of Boston University and Boston Medical Center, Boston, MA 02118, USA.
Pushpinder BawaCenter for Regenerative Medicine of Boston University and Boston Medical Center, Boston, MA 02118, USA.ORCID 0000-0002-8370-3638
Darrell N KottonCenter for Regenerative Medicine of Boston University and Boston Medical Center, Boston, MA 02118, USA.
Patrick L SinnUniversity of Iowa, Stead Family Department of Pediatrics, Iowa City, IA, 52245, USA.ORCID 0000-0002-9746-966X
Jennifer A WambachEdward Mallinckrodt Department of Pediatrics, Washington University in St. Louis, St. Louis Children's Hospital, St. Louis, MO 63110, USA.
Paul B McCrayUniversity of Iowa, Stead Family Department of Pediatrics, Iowa City, IA, 52245, USA.

Funding

Vector Core-Core 2P30DK054759 · NIDDK · UNIVERSITY OF IOWA · PI Alejandro Antonio Pezzulo · 1998 to 2026
$30.5M
Pathology CoreP01HL152960 · NHLBI · UNIVERSITY OF IOWA · PI ENGELHARDT, JOHN F · 2020 to 2024
$11.6M
Patient-specific iPSCs to model and treat the inception of pulmonary fibrosisP01HL170952 · NHLBI · BOSTON UNIVERSITY MEDICAL CAMPUS · PI Darrell N. Kotton · 2024 to 2026
$11.4M
Derivation of Transplantable Lung Epithelial Progenitors from iPS CellsR01HL095993 · NHLBI · BOSTON UNIVERSITY MEDICAL CAMPUS · PI Darrell N. Kotton · 2009 to 2026
$7.5M
Life-long phenotypic correction of CF airwaysR01HL133089 · NHLBI · UNIVERSITY OF IOWA · PI PATRICK L SINN · 2017 to 2026
$5.3M
NRSA Training CoreTL1TR001410 · NCATS · BOSTON UNIVERSITY MEDICAL CAMPUS · PI KOTTON, DARRELL N. · 2015 to 2024
$4.3M
Functional Characterization of ABCA3 Genomic VariantsR01HL149853 · NHLBI · WASHINGTON UNIVERSITY · PI Jennifer Wambach · 2020 to 2026
$3.9M
Adenine Base Edited Correction of Cystic Fibrosis AirwaysR01HL171035 · NHLBI · UNIVERSITY OF IOWA · PI PAUL B MCCRAY, PATRICK L SINN · 2024 to 2026
$1.9M
Impact of ATP-binding cassette A3 (ABCA3) mutations on alveolar type 2 cell function and genetic complementationR03TR004814 · NCATS · UNIVERSITY OF IOWA · PI MCCRAY, PAUL B · 2024 to 2024
$173k
NCATS NIH HHS R03 TR004814NCATS NIH HHS TL1 TR001410NHLBI NIH HHS P01 HL152960NHLBI NIH HHS P01 HL170952NHLBI NIH HHS R01 HL095993NHLBI NIH HHS R01 HL133089NHLBI NIH HHS R01 HL149853NHLBI NIH HHS R01 HL171035NIDDK NIH HHS P30 DK054759
6 · The paper itself

Abstract

The ATP-binding cassette subfamily A member 3 (ABCA3) protein on the limiting membrane of lamellar bodies in alveolar type 2 (AT2) cells transports phospholipids required for pulmonary surfactant assembly. ABCA3 deficiency results from biallelic pathogenic variants in

Indexed as

ABCA3 deficiencyalveolar epithelial type 2 cellsgene therapylamellar bodieslentiviral vectorssurfactant dysfunction

Identifiers

PMID40909633
PMCPMC12407927

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.