Evidence map›Paper›PMID 41614872›Full record

ArticleCurrent issues in molecular biology2025

Collagen Type II-Targeting Lentiviral Gene Therapy for Mucopolysaccharidosis IVA.

Betul Celik, Sampurna Saikia, Shaukat Khan, Krishna Sai Musini, Shunji Tomatsu

Abstract read
In one paragraph

Article in Current issues in molecular biology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing 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

2 citing papers in PubMed.

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

5 authors.

Betul CelikNemours Children's Health, 1600 Rockland Rd., Wilmington, DE 19803, USA.ORCID 0000-0002-0379-8076
Sampurna SaikiaNemours Children's Health, 1600 Rockland Rd., Wilmington, DE 19803, USA.ORCID 0009-0003-7994-2032
Shaukat KhanNemours Children's Health, 1600 Rockland Rd., Wilmington, DE 19803, USA.
Krishna Sai MusiniNemours Children's Health, 1600 Rockland Rd., Wilmington, DE 19803, USA.ORCID 0000-0002-0314-462X
Shunji TomatsuNemours Children's Health, 1600 Rockland Rd., Wilmington, DE 19803, USA.ORCID 0000-0002-0673-2160

Funding

Non-invasive functional assessment and pathogenesis of Morquio AR01HD102545 · NICHD · NEMOURS CHILDREN'S HOSPITAL, DELAWARE · PI TOMATSU, SHUNJI · 2021 to 2025
$2.9M
NICHD NIH HHS R01 HD102545NIH HHS 1R01HD102545-01A1the National MPS Society ID 4892the Turkish Ministry of National Education YLSY2017-Doctoral Scholarship
6 · The paper itself

Abstract

Mucopolysaccharidosis (MPS IVA) is caused by pathogenic variations in the

Indexed as

CBhCOL2A1collagen-type II targetingintravenous and intraarticular injectionlentiviral gene therapyMPS IVAnewborn treatmentWYRGRL peptide

Identifiers

PMID41614872
PMCPMC12839894

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.