Evidence map›Paper›PMID 39764351›Full record

ArticleMolecular therapy. Methods & clinical development2024

Human iPSC-derived neural stem cells engraft and improve pathophysiology of MPS I mice.

Caitlin C Calhoun, Shih-Hsin Kan, Alexander E Stover, Jerry F Harb, Edwin S Monuki, Raymond Y Wang, Philip H Schwartz

Abstract read
PubMed Full text at PMC Publisher
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 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. 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

7 authors.

Caitlin C CalhounResearch Institute, Children's Hospital of Orange County, Orange, CA, USA.
Shih-Hsin KanResearch Institute, Children's Hospital of Orange County, Orange, CA, USA.
Alexander E StoverResearch Institute, Children's Hospital of Orange County, Orange, CA, USA.
Jerry F HarbResearch Institute, Children's Hospital of Orange County, Orange, CA, USA.
Edwin S MonukiDepartment of Pathology and Laboratory Medicine, University of California, Irvine, Irvine, CA, USA.
Raymond Y WangCHOC Children's Specialists, Orange, CA, USA.
Philip H SchwartzResearch Institute, Children's Hospital of Orange County, Orange, CA, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Mucopolysaccharidosis type I (MPS I) is a metabolic disorder characterized by a deficiency in α-l-iduronidase (IDUA), leading to impaired glycosaminoglycan degradation. Current approved treatments seek to restore IDUA levels via enzyme replacement therapy (ERT) and/or hematopoietic stem cell transplantation (HSCT). The effectiveness of these treatment strategies in preventing neurodegeneration is limited due to the inability of ERT to penetrate the blood-brain barrier (BBB) and HSCT's limited CNS reconstitution of IDUA levels. We reprogrammed human cord blood cells into induced pluripotent stem cells (iPSCs), differentiated them into human induced neural stem cells (hiNSCs), and sorted them using fluorescence-activated cell sorting (FACS). Our

Indexed as

cell- and tissue-based therapycell differentiationCNSCXCR4hereditaryinduced pluripotent stem cellinflammationneural stem cellneurodegenerative diseasesNSC

Identifiers

PMID39764351
PMCPMC11701249

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.