Evidence map›Paper›PMID 32414402›Full record

ArticleStem cell research & therapy2020

Generation of gene-corrected functional osteoclasts from osteopetrotic induced pluripotent stem cells.

Xiaojie Xian, Roksana Moraghebi, Henrik Löfvall, Anders Fasth, Kim Henriksen, Johan Richter, Niels-Bjarne Woods, Ilana Moscatelli

Open access · goldAbstract read
In one paragraph

Article in Stem cell research & therapy, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.

0numbers the graph read from it
0cells of the map it votes in
11citing papers in PubMed
1.1field-weighted citation impact, top 24% of its field
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

11 citing papers in PubMed, 15 citations in OpenAlex.

  1. Review
  2. Article
  3. Article
  4. Review
  5. Article
  6. Article
  7. Review
  8. Review
  9. Review
  10. Review
  11. 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

8 authors at 3 institutions in 2 countries.

Xiaojie XianDepartment of Molecular Medicine and Gene Therapy, Lund Strategic Center for Stem Cell Biology, Lund University, BMC A12, 221 84, Lund, Sweden.
Roksana MoraghebiDepartment of Molecular Medicine and Gene Therapy, Lund Strategic Center for Stem Cell Biology, Lund University, BMC A12, 221 84, Lund, Sweden.
Henrik LöfvallDepartment of Molecular Medicine and Gene Therapy, Lund Strategic Center for Stem Cell Biology, Lund University, BMC A12, 221 84, Lund, Sweden.
Anders FasthDepartment of Pediatrics, Institute of Clinical Sciences, University of Gothenburg, Gothenburg, Sweden.
Kim HenriksenNordic Bioscience, Herlev, Denmark.
Johan RichterDepartment of Molecular Medicine and Gene Therapy, Lund Strategic Center for Stem Cell Biology, Lund University, BMC A12, 221 84, Lund, Sweden.
Niels-Bjarne WoodsDepartment of Molecular Medicine and Gene Therapy, Lund Strategic Center for Stem Cell Biology, Lund University, BMC A12, 221 84, Lund, Sweden.
Ilana MoscatelliDepartment of Molecular Medicine and Gene Therapy, Lund Strategic Center for Stem Cell Biology, Lund University, BMC A12, 221 84, Lund, Sweden. Ilana.Moscatelli@med.lu.se.ORCID 0000-0003-0956-2053
Lund University · SENordic Bioscience (Denmark) · DKUniversity of Gothenburg · SE

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundInfantile malignant osteopetrosis (IMO) is an autosomal recessive disorder characterized by non-functional osteoclasts and a fatal outcome early in childhood. About 50% of patients have mutations in the TCIRG1 gene.

methodsIMO iPSCs were generated from a patient carrying a homozygous c.11279G>A (IVS18+1) mutation in TCIRG1 and transduced with a lentiviral vector expressing human TCIRG1. Embryoid bodies were generated and differentiated into monocytes. Non-adherent cells were harvested and further differentiated into osteoclasts on bovine bone slices.

resultsRelease of the bone resorption biomarker CTX-I into the media of gene-corrected osteoclasts was 5-fold higher than that of the uncorrected osteoclasts and 35% of that of control osteoclasts. Bone resorption potential was confirmed by the presence of pits on the bones cultured with gene-corrected osteoclasts, absent in the uncorrected IMO osteoclasts.

conclusionsThe disease phenotype was partially corrected in vitro, providing a valuable resource for therapy development for this form of severe osteopetrosis.

Indexed as

Bone ResorptionInduced Pluripotent Stem CellsOsteopetrosisVacuolar Proton-Translocating ATPasesAnimalsCattleHumansMutationOsteoclastsTCIRG1 protein, humanVacuolar Proton-Translocating ATPasesGene transferInfantile malignant osteopetrosisiPSC generationOsteoclast differentiation

Identifiers

PMID32414402
PMCPMC7227215
OpenAlexW3024314900

What Socratic holds

Textmetadata
LicenceCC BY
Read underepoch 390

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 Socratic graph.