Evidence map›Paper›PMID 39391765›Full record

ArticleMolecular therapy. Nucleic acids2024

Novel readthrough agent suppresses nonsense mutations and restores functional type VII collagen and laminin 332 in epidermolysis bullosa.

Brandon Levian, Yingping Hou, Xin Tang, Liat Bainvoll, Kate Zheng, Vasu Badarinarayana, Soheil Aghamohammadzadeh, Mei Chen

Abstract read
In one paragraph

Article in Molecular therapy. Nucleic acids, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed, 1 pooled it
–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

8 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Distinct responses ofMolecular therapy. Nucleic acids · 2026
    Article
  3. RiboScreenBiomedicines · 2026
    Review
  4. Article
  5. Treatment Approaches for Alport Syndrome.Journal of the American Society of Nephrology : JASN · 2026
    Review
  6. Review
  7. Article
  8. 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

8 authors.

Brandon LevianDepartment of Dermatology, The Keck School of Medicine, University of Southern California, Los Angeles, CA 90033, USA.
Yingping HouDepartment of Dermatology, The Keck School of Medicine, University of Southern California, Los Angeles, CA 90033, USA.
Xin TangDepartment of Dermatology, The Keck School of Medicine, University of Southern California, Los Angeles, CA 90033, USA.
Liat BainvollDepartment of Dermatology, The Keck School of Medicine, University of Southern California, Los Angeles, CA 90033, USA.
Kate ZhengDepartment of Dermatology, The Keck School of Medicine, University of Southern California, Los Angeles, CA 90033, USA.
Vasu BadarinarayanaEloxx Pharmaceuticals, Watertown, MA, USA.
Soheil AghamohammadzadehEloxx Pharmaceuticals, Watertown, MA, USA.
Mei ChenDepartment of Dermatology, The Keck School of Medicine, University of Southern California, Los Angeles, CA 90033, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Recessive dystrophic epidermolysis bullosa (RDEB) and junctional epidermolysis bullosa (JEB) are lethal blistering skin disorders resulting from mutations in genes coding for type VII collagen (

Indexed as

epidermolysis bullosaextracellular matrixgenetic diseasesMT: Delivery Strategiesreadthrough therapyskin

Identifiers

PMID39391765
PMCPMC11465179

What OpenQuestion holds

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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.