Evidence map›Paper›PMID 40441149›Full record

ArticleMolecular therapy : the journal of the American Society of Gene Therapy2025

Lung endothelial transduction in a patient that succumbed to acute respiratory distress syndrome following high-dose rAAV9 gene therapy.

Juliette Hordeaux, R Jason Lamontagne, Sushobhana Bandyopadhyay, Peter Bell, James M Wilson, Terence R Flotte

Abstract read
In one paragraph

Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing 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

6 citing papers in PubMed.

  1. Trial
  2. Death following high-dose AAV9 gene therapy in a patient with advanced SMA-PME.Molecular therapy : the journal of the American Society of Gene Therapy · 2026
    Article
  3. Review
  4. Immune Toxicities in AAV Gene Therapy: Overview for Clinicians.International journal of molecular sciences · 2026
    Review
  5. Article
  6. 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

6 authors.

Juliette HordeauxGemma Biotherapeutics, Inc., Philadelphia, PA, USA. Electronic address: juliette.hordeaux@gemmabiotx.com.
R Jason LamontagneGemma Biotherapeutics, Inc., Philadelphia, PA, USA.
Sushobhana BandyopadhyayDepartment of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Peter BellGemma Biotherapeutics, Inc., Philadelphia, PA, USA.
James M WilsonGemma Biotherapeutics, Inc., Philadelphia, PA, USA; Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Terence R FlotteHorae Gene Therapy Center and the Li Weibo Institute for Rare Diseases Research, University of Massachusetts Chan Medical School, Worcester, MA, USA. Electronic address: terry.flotte@umassmed.edu.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

PubMed holds no abstract for this paper.

Identifiers

PMID40441149
PMCPMC12172266

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.