Evidence map›Paper›PMID 35011669›Full record

ReviewCells2021

Stem Cell-Based Disease Models for Inborn Errors of Immunity.

Aline Zbinden, Kirsten Canté-Barrett, Karin Pike-Overzet, Frank J T Staal

Abstract readReview
In one paragraph

Review in Cells, 2021. 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

4 authors.

Aline ZbindenDepartment of Immunology, Leiden University Medical Center, 2333 ZA Leiden, The Netherlands.
Kirsten Canté-BarrettDepartment of Immunology, Leiden University Medical Center, 2333 ZA Leiden, The Netherlands.ORCID 0000-0003-0418-8445
Karin Pike-OverzetDepartment of Immunology, Leiden University Medical Center, 2333 ZA Leiden, The Netherlands.
Frank J T StaalDepartment of Immunology, Leiden University Medical Center, 2333 ZA Leiden, The Netherlands.ORCID 0000-0003-1588-8519

Funding

Swiss National Science Foundation P500PB_203146 / 1
6 · The paper itself

Abstract

The intrinsic capacity of human hematopoietic stem cells (hHSCs) to reconstitute myeloid and lymphoid lineages combined with their self-renewal capacity hold enormous promises for gene therapy as a viable treatment option for a number of immune-mediated diseases, most prominently for inborn errors of immunity (IEI). The current development of such therapies relies on disease models, both in vitro and in vivo, which allow the study of human pathophysiology in great detail. Here, we discuss the current challenges with regards to developmental origin, heterogeneity and the subsequent implications for disease modeling. We review models based on induced pluripotent stem cell technology and those relaying on use of adult hHSCs. We critically review the advantages and limitations of current models for IEI both in vitro and in vivo. We conclude that existing and future stem cell-based models are necessary tools for developing next generation therapies for IEI.

Indexed as

HumansImmune System DiseasesMetabolism, Inborn ErrorsStem Cellshematopoietic stem cellsimmune deficiencyinborn errors of immunityinduced pluripotent stem cellsprimary immune deficiency

Identifiers

PMID35011669
PMCPMC8750661

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.