Evidence map›Paper›PMID 36166236›Full record

ReviewHuman gene therapy2022

Gene Therapeutics for Surfactant Dysfunction Disorders: Targeting the Alveolar Type 2 Epithelial Cell.

Sneha Sitaraman, Konstantinos-Dionysios Alysandratos, Jennifer A Wambach, Maria P Limberis

Open access · greenAbstract readReview
In one paragraph

Review in Human gene therapy, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
18citing papers in PubMed, 1 pooled it
2.1field-weighted citation impact, top 11% of its field
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

18 citing papers in PubMed, 1 synthesis or guideline pooled it, 17 citations in OpenAlex.

  1. Old and emerging therapies for childhood interstitial lung disease (chILD): a systematic review.European respiratory review : an official journal of the European Respiratory Society · 2026
    Pooled it
  2. Article
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  5. Biallelic LAMP3 variants in 5 families with interstitial lung disease: Evidence of a disease-gene association.Genetics in medicine : official journal of the American College of Medical Genetics · 2026
    Article
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  7. Article
  8. Review
  9. Article
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  11. Article
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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

4 authors at 3 institutions in 1 country.

Sneha SitaramanSpirovant Sciences, Inc., Philadelphia, Pennsylvania, USA.ORCID 0000-0001-8541-9190
Konstantinos-Dionysios AlysandratosCenter for Regenerative Medicine, Boston University and Boston Medical Center, Boston, Massachusetts, USA.
Jennifer A WambachDivision of Newborn Medicine, Edward Mallinckrodt Department of Pediatrics, Washington University School of Medicine and St. Louis Children's Hospital, St. Louis, Missouri, USA.
Maria P LimberisSpirovant Sciences, Inc., Philadelphia, Pennsylvania, USA.ORCID 0000-0001-8157-7490
Roivant Sciences (United States) · USBoston University · USSt. Louis Children's Hospital · US

Funding

Modeling, pathogenesis and treatment of idiopathic pulmonary fibrosisU01HL134760 · NHLBI · COLUMBIA UNIVERSITY HEALTH SCIENCES · PI SNOECK, HANS-WILLEM E, VUNJAK-NOVAKOVIC, GORDANA · 2016 to 2022
$8.5M
Functional Characterization of ABCA3 Genomic VariantsR01HL149853 · NHLBI · WASHINGTON UNIVERSITY · PI Jennifer Wambach · 2020 to 2026
$3.9M
NHLBI NIH HHS R01 HL149853NHLBI NIH HHS U01 HL134760
6 · The paper itself

Abstract

Genetic disorders of surfactant dysfunction result in significant morbidity and mortality, among infants, children, and adults. Available medical interventions are limited, nonspecific, and generally ineffective. As such, the need for effective therapies remains. Pathogenic variants in the

Indexed as

Lung Diseases, InterstitialPulmonary SurfactantsAdultAlveolar Epithelial CellsChildEpithelial CellsHumansInfantLungMutationPulmonary SurfactantsABCA3alveolar type 2 epithelial cellAT2gene therapypulmonary surfactantSFTPBSFTPCviral vectors

Identifiers

PMID36166236
PMCPMC9595619
OpenAlexW4297264964

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.