Evidence map›Paper›PMID 39317061›Full record

ArticleStem cell research2024

Six induced pluripotent stem cell lines from fibroblasts of individuals with CLN3-related conditions.

Ewelina Dwojak, Danielle O'Mard, Jizhong Zou, Christopher A Wassif, Sandra Burkett, Michael Eckhaus, Fabio Rueda Faucz, Cameron Padilla, Rafael Villasmil, Wei Zheng and 1 more

Abstract read
In one paragraph

Article in Stem cell research, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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

1 citing paper in PubMed.

  1. 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

11 authors.

Ewelina DwojakUnit on Cellular Stress in Development and Diseases, Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD, USA.
Danielle O'MardUnit on Cellular Stress in Development and Diseases, Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD, USA.
Jizhong ZouiPSC Core, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD, USA.
Christopher A WassifSection on Molecular Dysmorphology, Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD, USA.
Sandra BurkettMolecular Cytogenetics Core Facility, National Cancer Institute, Frederick, MD, USA.
Michael EckhausDiagnostic and Research Services Branch, Office of the Director, National Institutes of Health, Bethesda, MD, USA.
Fabio Rueda FauczMolecular Genomics Core, Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD, USA.
Cameron PadillaMolecular Genomics Core, Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD, USA.
Rafael VillasmilFlow Cytometry Core Facility, National Eye Institute, Bethesda, MD, USA.
Wei ZhengTherapeutic Development Branch, National Center for Advancing Translational Sciences, National Institutes of Health, Bethesda, MD, USA.
An N Dang DoUnit on Cellular Stress in Development and Diseases, Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD, USA. Electronic address: an.dangdo@nih.gov.

Funding

Drug Repurposing Screening for Rare and Neglected DiseasesZIATR000018 · NCATS · NATIONAL CENTER FOR ADVANCING TRANSLATIONAL SCIENCES · PI ZHENG, WEI · 2015 to 2025
$24.6M
Research Investigations of Cellular Stress in Development and DiseasesZIAHD009001 · NICHD · EUNICE KENNEDY SHRIVER NATIONAL INSTITUTE OF CHILD HEALTH & HUMAN DEVELOPMENT · PI DANG DO, AN · 2022 to 2025
$4.9M
Intramural NIH HHS ZIA HD009001Intramural NIH HHS ZIA TR000018
6 · The paper itself

Abstract

Primary fibroblasts from six individuals with CLN3-related conditions were used to generate induced pluripotent stem cell (iPSC) lines CHDTRi001-B, CHDTRi002-B, CHDTRi003-A, CHDTRi004-B, CHDTRi005-A, and CHDTRi006-E through the expression of four reprogramming factors: human OCT3/4, KLF4, SOX2, and c-MYC. The iPSC lines were characterized to confirm their pluripotency via immunocytochemistry, flow cytometry, and teratoma formation. Genomic stability, cell line identity, and CLN3 genotype were confirmed. These iPSC lines may be used as participant-derived experimental models for further investigation of CLN3, a rare, fatal, pediatric, blindness and neurodegenerative lysosomal disorder with no cure.

Indexed as

FibroblastsInduced Pluripotent Stem CellsKruppel-Like Factor 4Cell DifferentiationCell LineCellular ReprogrammingFemaleHumansMaleMembrane GlycoproteinsMolecular ChaperonesCLN3 protein, humanKLF4 protein, humanKruppel-Like Factor 4Membrane GlycoproteinsMolecular Chaperones

Identifiers

PMID39317061
PMCPMC11645225

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.