Evidence map›Paper›PMID 37805711›Full record

ArticleMolecular therapy : the journal of the American Society of Gene Therapy2023

A promoterless AAV6.2FF-based lung gene editing platform for the correction of surfactant protein B deficiency.

Sylvia P Thomas, Jakob M Domm, Jacob P van Vloten, Liqun Xu, Arul Vadivel, Jacob G E Yates, Yanlong Pei, Joelle Ingrao, Laura P van Lieshout, Sergio R Jackson and 11 more

Open access · bronzeAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.

0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed
2.2field-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

8 citing papers in PubMed, 7 citations in OpenAlex.

  1. Article
  2. International journal of biological sciences · 2026
    Review
  3. Article
  4. Article
  5. THERAPIES FOR NEONATAL DISEASES OF THE SURFACTANT SYSTEM.Transactions of the American Clinical and Climatological Association · 2025
    Review
  6. Review
  7. Moving on from clinical animal-derived surfactants to peptide-based synthetic pulmonary surfactant.American journal of physiology. Lung cellular and molecular physiology · 2024
    Review
  8. In vivo genome editing shows promise for treating pulmonary diseases.Molecular therapy : the journal of the American Society of Gene Therapy · 2023
    Article
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

21 authors at 4 institutions in 2 countries.

Sylvia P ThomasDepartment of Pathobiology, University of Guelph, Guelph, ON N1G 2W1, Canada.
Jakob M DommDepartment of Pathobiology, University of Guelph, Guelph, ON N1G 2W1, Canada.
Jacob P van VlotenDepartment of Pathobiology, University of Guelph, Guelph, ON N1G 2W1, Canada.
Liqun XuRegenerative Medicine Program, The Ottawa Hospital Research Institute (OHRI), Ottawa, ON, Canada; Department of Cellular and Molecular Medicine, University of Ottawa, Ottawa, ON, Canada; Neonatology, Department of Pediatrics, Children's Hospital of Eastern Ontario (CHEO), and CHEO Research Institute, Ottawa, ON K1Y 4E9, Canada.
Arul VadivelRegenerative Medicine Program, The Ottawa Hospital Research Institute (OHRI), Ottawa, ON, Canada; Department of Cellular and Molecular Medicine, University of Ottawa, Ottawa, ON, Canada; Neonatology, Department of Pediatrics, Children's Hospital of Eastern Ontario (CHEO), and CHEO Research Institute, Ottawa, ON K1Y 4E9, Canada.
Jacob G E YatesDepartment of Pathobiology, University of Guelph, Guelph, ON N1G 2W1, Canada.
Yanlong PeiDepartment of Pathobiology, University of Guelph, Guelph, ON N1G 2W1, Canada.
Joelle IngraoDepartment of Pathobiology, University of Guelph, Guelph, ON N1G 2W1, Canada.
Laura P van LieshoutDepartment of Pathobiology, University of Guelph, Guelph, ON N1G 2W1, Canada.
Sergio R JacksonDepartment of Biomedical Sciences, University of Pennsylvania School of Veterinary Medicine, Philadelphia, PA 19104, USA.
Jessica A MinottDepartment of Pathobiology, University of Guelph, Guelph, ON N1G 2W1, Canada.
Adithya AchuthanRegenerative Medicine Program, The Ottawa Hospital Research Institute (OHRI), Ottawa, ON, Canada; Department of Cellular and Molecular Medicine, University of Ottawa, Ottawa, ON, Canada; Neonatology, Department of Pediatrics, Children's Hospital of Eastern Ontario (CHEO), and CHEO Research Institute, Ottawa, ON K1Y 4E9, Canada.
Yeganeh MehraniDepartment of Pathobiology, University of Guelph, Guelph, ON N1G 2W1, Canada.
Thomas M McAuslandDepartment of Pathobiology, University of Guelph, Guelph, ON N1G 2W1, Canada.
Wei ZhangDepartment of Molecular and Cellular Biology, University of Guelph, Guelph, ON, Canada.
Khalil KarimiDepartment of Pathobiology, University of Guelph, Guelph, ON N1G 2W1, Canada.
Andrew E VaughanDepartment of Biomedical Sciences, University of Pennsylvania School of Veterinary Medicine, Philadelphia, PA 19104, USA.
Jondavid de JongDepartment of Pathobiology, University of Guelph, Guelph, ON N1G 2W1, Canada.
Martin H KangDepartment of Pediatrics, Darby Children's Research Institute, Medical University of South Carolina, Charleston, SC 29425, USA.
Bernard ThebaudRegenerative Medicine Program, The Ottawa Hospital Research Institute (OHRI), Ottawa, ON, Canada; Department of Cellular and Molecular Medicine, University of Ottawa, Ottawa, ON, Canada; Neonatology, Department of Pediatrics, Children's Hospital of Eastern Ontario (CHEO), and CHEO Research Institute, Ottawa, ON K1Y 4E9, Canada.
Sarah K WoottonDepartment of Pathobiology, University of Guelph, Guelph, ON N1G 2W1, Canada. Electronic address: kwootton@uoguelph.ca.
University of Guelph · CAChildren's Hospital of Eastern Ontario · CAUniversity of Pennsylvania · USMedical University of South Carolina · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Surfactant protein B (SP-B) deficiency is a rare genetic disease that causes fatal respiratory failure within the first year of life. Currently, the only corrective treatment is lung transplantation. Here, we co-transduced the murine lung with adeno-associated virus 6.2FF (AAV6.2FF) vectors encoding a SaCas9-guide RNA nuclease or donor template to mediate insertion of promoterless reporter genes or the (murine) Sftpb gene in frame with the endogenous surfactant protein C (SP-C) gene, without disrupting SP-C expression. Intranasal administration of 3 × 10

Indexed as

DoxycyclineGene EditingAnimalsCRISPR-Cas SystemsDependovirusGenetic VectorsLungLung Diseases, InterstitialMicePulmonary Alveolar ProteinosisPulmonary Surfactant-Associated Protein BRNA, Guide, CRISPR-Cas SystemsSurface-Active AgentsDoxycyclinePulmonary Surfactant-Associated Protein BRNA, Guide, CRISPR-Cas SystemsSurface-Active AgentsAAVAAV6.2FFadeno-associated virusalveolar type II cellsCRISPR-Cas9gene editinggene rideHDRhomology directed repairintranasalsurfactant protein B deficiencysurfactant protein C

Identifiers

PMID37805711
PMCPMC10727957
OpenAlexW4387424145

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.