Evidence map›Paper›PMID 31530844›Full record

ArticleScientific reports2019

Reversal of Surfactant Protein B Deficiency in Patient Specific Human Induced Pluripotent Stem Cell Derived Lung Organoids by Gene Therapy.

Sandra Lawrynowicz Leibel, Alicia Winquist, Irene Tseu, Jinxia Wang, Daochun Luo, Sharareh Shojaie, Neal Nathan, Evan Snyder, Martin Post

Open access · goldAbstract read
In one paragraph

Article in Scientific reports, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 49 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
49citing papers in PubMed, 2 pooled it
7.0field-weighted citation impact, top 2% 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

49 citing papers in PubMed, 2 syntheses or guidelines pooled it, 76 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. Pooled it
  3. Review
  4. Article
  5. Human lung organoids model for assessing host response toFrontiers in cellular and infection microbiology · 2026
    Article
  6. Lung organoids: a new frontier in neonatology and paediatric respiratory medicine.European respiratory review : an official journal of the European Respiratory Society · 2025
    Review
  7. Review
  8. Review
  9. Article
  10. Article
  11. THERAPIES FOR NEONATAL DISEASES OF THE SURFACTANT SYSTEM.Transactions of the American Clinical and Climatological Association · 2025
    Review
  12. Advancements in theFrontiers in toxicology · 2025
    Review
  13. NAM journal · 2025
    Article
  14. Article
  15. A therapy for suppressing canonical and noncanonical SARS-CoV-2 viral entry and an intrinsic intrapulmonary inflammatory response.Proceedings of the National Academy of Sciences of the United States of America · 2024
    Article
  16. Review
  17. Article
  18. Review
  19. Article
  20. 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

9 authors at 5 institutions in 2 countries.

Sandra Lawrynowicz LeibelDepartment of Pediatrics, University of California, San Diego, Rady Children's Hospital, San Diego, La Jolla, CA, USA. saleibel@ucsd.edu.
Alicia WinquistSanford Burnham Prebys Medical Discovery Institute, La Jolla, CA, USA.
Irene TseuTranslational Medicine Program, Peter Gilgan Centre for Research and Learning, Hospital for Sick Children, Toronto, Ontario, Canada.
Jinxia WangTranslational Medicine Program, Peter Gilgan Centre for Research and Learning, Hospital for Sick Children, Toronto, Ontario, Canada.
Daochun LuoTranslational Medicine Program, Peter Gilgan Centre for Research and Learning, Hospital for Sick Children, Toronto, Ontario, Canada.
Sharareh ShojaieTranslational Medicine Program, Peter Gilgan Centre for Research and Learning, Hospital for Sick Children, Toronto, Ontario, Canada.
Neal NathanSanford Burnham Prebys Medical Discovery Institute, La Jolla, CA, USA.
Evan SnyderDepartment of Pediatrics, University of California, San Diego, Rady Children's Hospital, San Diego, La Jolla, CA, USA.
Martin PostTranslational Medicine Program, Peter Gilgan Centre for Research and Learning, Hospital for Sick Children, Toronto, Ontario, Canada.
Hospital for Sick Children · CASanford Burnham Prebys Medical Discovery Institute · USSanford Consortium for Regenerative Medicine · USUniversity of California, San Diego · USUniversity of Toronto · CA

Funding

CIHR FDN-143309
6 · The paper itself

Abstract

Surfactant protein B (SFTPB) deficiency is a fatal disease affecting newborn infants. Surfactant is produced by alveolar type II cells which can be differentiated in vitro from patient specific induced pluripotent stem cell (iPSC)-derived lung organoids. Here we show the differentiation of patient specific iPSCs derived from a patient with SFTPB deficiency into lung organoids with mesenchymal and epithelial cell populations from both the proximal and distal portions of the human lung. We alter the deficiency by infecting the SFTPB deficient iPSCs with a lentivirus carrying the wild type SFTPB gene. After differentiating the mutant and corrected cells into lung organoids, we show expression of SFTPB mRNA during endodermal and organoid differentiation but the protein product only after organoid differentiation. We also show the presence of normal lamellar bodies and the secretion of surfactant into the cell culture medium in the organoids of lentiviral infected cells. These findings suggest that a lethal lung disease can be targeted and corrected in a human lung organoid model in vitro.

Indexed as

Genetic TherapyInduced Pluripotent Stem CellsLungPulmonary Alveolar ProteinosisPulmonary Surfactant-Associated Protein BCell DifferentiationEpithelial CellsFibroblastsGenetic MarkersGreen Fluorescent ProteinsHumansLentivirusLung Diseases, InterstitialOrganoidsPulmonary AlveoliGenetic MarkersGreen Fluorescent ProteinsPulmonary Surfactant-Associated Protein B

Identifiers

PMID31530844
PMCPMC6748939
OpenAlexW2973363745

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.