Evidence map›Paper›PMID 40677837›Full record

ArticleMolecular therapy. Methods & clinical development2025

Betul Celik, Estera Rintz, Shaukat Khan, Andrés Felipe Leal, Fnu Nidhi, Shunji Tomatsu

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Methods & clinical development, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing 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

4 citing papers in PubMed.

  1. Article
  2. Advances in Therapies for Mucopolysaccharidoses.Current issues in molecular biology · 2026
    Review
  3. Natural History of Morquio A Syndrome.Journal of inherited metabolic disease · 2026
    Review
  4. 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

6 authors.

Betul CelikDepartment of Biological Sciences, University of Delaware, Newark, DE 19716, USA.
Estera RintzDepartment of Molecular Biology, Faculty of Biology, University of Gdansk, 80-308 Gdansk, Poland.
Shaukat KhanSkeletal Dysplasia Research Lab, Nemours Children's Health, Wilmington, DE 19803, USA.
Andrés Felipe LealSkeletal Dysplasia Research Lab, Nemours Children's Health, Wilmington, DE 19803, USA.
Fnu NidhiDepartment of Biological Sciences, University of Delaware, Newark, DE 19716, USA.
Shunji TomatsuDepartment of Biological Sciences, University of Delaware, Newark, DE 19716, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Mucopolysaccharidosis IVA (MPS IVA) is an autosomal recessive disorder that causes the accumulation of keratan sulfate (KS) and chondroitin-6-sulfate in bone and cartilage. This results in progressive skeletal dysplasia, with no effective treatment available. Our study hypothesized that direct lentiviral vector (LV) gene therapy could produce active enzymes from transduced cells, impacting bone and cartilage lesions in MPS IVA. We developed LVs carrying the

Indexed as

CBhCD11bCOL2A1intravenous-intraarticular-intramuscular injectionlentiviral gene therapyMPS IVAnewborn treatment

Identifiers

PMID40677837
PMCPMC12269278

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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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.