Evidence map›Paper›PMID 38638546›Full record

ArticleMolecular therapy. Methods & clinical development2024

Durable transgene expression and efficient re-administration after rAAV2.5T-mediated fCFTRΔR gene delivery to adult ferret lungs.

Yinghua Tang, Mehrnoosh Ebadi, Junying Lei, Zehua Feng, Shahab Fakhari, Peipei Wu, Mark D Smith, Maria P Limberis, Roland Kolbeck, Katherine J Excoffon and 2 more

Open access · goldAbstract read
In one paragraph

Article in Molecular therapy. Methods & clinical development, 2024. 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
2.8field-weighted citation impact, top 10% 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, 5 citations in OpenAlex.

  1. Molecular therapy. Nucleic acids · 2026
    Article
  2. Review
  3. Review
  4. Review
  5. Review
  6. Article
  7. Nonclinical strategies and considerations to enable the redosing of gene therapies.Molecular therapy. Methods & clinical development · 2025
    Review
  8. Humoral and cellular immune responses to AAV delivery in the airway.Molecular therapy. Methods & clinical development · 2024
    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

12 authors at 1 institution in 1 country.

Yinghua TangDepartment of Anatomy & Cell Biology, University of Iowa Carver College of Medicine, Iowa City, IA 52242, USA.
Mehrnoosh EbadiDepartment of Anatomy & Cell Biology, University of Iowa Carver College of Medicine, Iowa City, IA 52242, USA.
Junying LeiDepartment of Anatomy & Cell Biology, University of Iowa Carver College of Medicine, Iowa City, IA 52242, USA.
Zehua FengDepartment of Anatomy & Cell Biology, University of Iowa Carver College of Medicine, Iowa City, IA 52242, USA.
Shahab FakhariDepartment of Anatomy & Cell Biology, University of Iowa Carver College of Medicine, Iowa City, IA 52242, USA.
Peipei WuDepartment of Anatomy & Cell Biology, University of Iowa Carver College of Medicine, Iowa City, IA 52242, USA.
Mark D SmithSpirovant Science Inc., Philadelphia, PA 19104, USA.
Maria P LimberisSpirovant Science Inc., Philadelphia, PA 19104, USA.
Roland KolbeckSpirovant Science Inc., Philadelphia, PA 19104, USA.
Katherine J ExcoffonSpirovant Science Inc., Philadelphia, PA 19104, USA.
Ziying YanDepartment of Anatomy & Cell Biology, University of Iowa Carver College of Medicine, Iowa City, IA 52242, USA.
John F EngelhardtDepartment of Anatomy & Cell Biology, University of Iowa Carver College of Medicine, Iowa City, IA 52242, USA.
University of Iowa · US

Funding

Pulmonary Toxicology Facility CoreP30ES005605 · NIEHS · UNIVERSITY OF IOWA · PI Jong Sung Kim · 1990 to 2026
$40.5M
Vector Core-Core 2P30DK054759 · NIDDK · UNIVERSITY OF IOWA · PI Alejandro Antonio Pezzulo · 1998 to 2026
$30.5M
Pathology CoreP01HL152960 · NHLBI · UNIVERSITY OF IOWA · PI WELSH, MICHAEL J. · 2020 to 2024
$11.6M
Biology of Submucosal Gland Stem Cells in the AirwayR01HL165404 · NHLBI · UNIVERSITY OF IOWA · PI ENGELHARDT, JOHN F · 2022 to 2025
$2.9M
NATIONAL FERRET RESOURCE AND RESEARCH CENTER ON LUNG DISEASE75N92019C00010 · NHLBI · UNIVERSITY OF IOWA · PI BOYLE, JESSICA · 2023 to 2023
$1.1M
NHLBI NIH HHS 75N92019C00010NHLBI NIH HHS P01 HL152960NHLBI NIH HHS R01 HL165404NIDDK NIH HHS P30 DK054759NIEHS NIH HHS P30 ES005605
6 · The paper itself

Abstract

The dosing interval for effective recombinant adeno-associated virus (rAAV)-mediated gene therapy of cystic fibrosis lung disease remains unknown. Here, we assessed the durability of rAAV2.5T-fCFTRΔR-mediated transgene expression and neutralizing antibody (NAb) responses in lungs of adult wild-type ferrets. Within the first 3 months following rAAV2.5T-fCFTRΔR delivery to the lung,

Indexed as

adeno-associated virusCFTRcystic fibrosisdurabilitygene therapylungneutralizing antibodiesrepeat administrationT cell responsestransgene

Identifiers

PMID38638546
PMCPMC11024656
OpenAlexW4393254325

What Socratic 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 Socratic graph.