Evidence map›Paper›PMID 25772073›Full record

ArticleCell stem cell2015

Lymphoid regeneration from gene-corrected SCID-X1 subject-derived iPSCs.

Tushar Menon, Amy L Firth, Deirdre D Scripture-Adams, Zoran Galic, Susan J Qualls, William B Gilmore, Eugene Ke, Oded Singer, Leif S Anderson, Alexander R Bornzin and 3 more

Open access · bronzeAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Cell stem cell, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 47 papers.

0numbers the graph read from it
0cells of the map it votes in
47citing papers in PubMed
5.0field-weighted citation impact, top 3% 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

47 citing papers in PubMed, 69 citations in OpenAlex.

  1. Article
  2. Article
  3. Review
  4. Article
  5. Review
  6. Basic Science Perspective on Engineering and Modeling the Large Airways.Advances in experimental medicine and biology · 2023
    Article
  7. Article
  8. Review
  9. Article
  10. Review
  11. Frontiers in immunology · 2022
    Review
  12. Article
  13. Review
  14. Review
  15. [Gene editing for the treatment of primary immunodeficiency disease].Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics · 2021
    Review
  16. Review
  17. Article
  18. Review
  19. Review
  20. Stem cells and lung regeneration.American journal of physiology. Cell physiology · 2020
    Review
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

13 authors at 4 institutions in 2 countries.

Tushar MenonThe Salk Institute of Biological Studies, Laboratory of Genetics, 10010 North Torrey Pines Road, La Jolla, CA 92037, USA.
Amy L FirthThe Salk Institute of Biological Studies, Laboratory of Genetics, 10010 North Torrey Pines Road, La Jolla, CA 92037, USA.
Deirdre D Scripture-AdamsDavid Geffen School of Medicine at UCLA, University of California, Los Angeles, 10833 Le Conte Avenue, Los Angeles, CA 90095-1678; UCLA AIDS Institute, University of California, Los Angeles, 10833 Le Conte Avenue, Los Angeles, CA 90095-1678.
Zoran GalicDavid Geffen School of Medicine at UCLA, University of California, Los Angeles, 10833 Le Conte Avenue, Los Angeles, CA 90095-1678; Broad Stem Cell Research Center, University of California, Los Angeles, 10833 Le Conte Avenue, Los Angeles, CA 90095-1678.
Susan J QuallsThe Salk Institute of Biological Studies, Laboratory of Genetics, 10010 North Torrey Pines Road, La Jolla, CA 92037, USA.
William B GilmoreThe Salk Institute of Biological Studies, Laboratory of Genetics, 10010 North Torrey Pines Road, La Jolla, CA 92037, USA.
Eugene KeThe Salk Institute of Biological Studies, Laboratory of Genetics, 10010 North Torrey Pines Road, La Jolla, CA 92037, USA.
Oded SingerThe Salk Institute of Biological Studies, Laboratory of Genetics, 10010 North Torrey Pines Road, La Jolla, CA 92037, USA.
Leif S AndersonThe Salk Institute of Biological Studies, Laboratory of Genetics, 10010 North Torrey Pines Road, La Jolla, CA 92037, USA.
Alexander R BornzinThe Salk Institute of Biological Studies, Laboratory of Genetics, 10010 North Torrey Pines Road, La Jolla, CA 92037, USA.
Ian E AlexanderGene Therapy Research Unit, Children's Medical Research Institute and The Children's Hospital at Westmead, Wentworthville, NSW 2145, Australia.
Jerome A ZackDepartment of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, 10833 Le Conte Avenue, Los Angeles, CA 90095-1678; David Geffen School of Medicine at UCLA, University of California, Los Angeles, 10833 Le Conte Avenue, Los Angeles, CA 90095-1678; UCLA AIDS Institute, University of California, Los Angeles, 10833 Le Conte Avenue, Los Angeles, CA 90095-1678; Broad Stem Cell Research Center, University of California, Los Angeles, 10833 Le Conte Avenue, Los Angeles, CA 90095-1678.
Inder M VermaThe Salk Institute of Biological Studies, Laboratory of Genetics, 10010 North Torrey Pines Road, La Jolla, CA 92037, USA. Electronic address: verma@salk.edu.
Salk Institute for Biological Studies · USBroad Center · USBlack AIDS Institute · USChildren's Hospital at Westmead · AU

Funding

Women's CancersP30CA016042 · NCI · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI Samuel Wheeler French · 1985 to 2026
$134.5M
Viral Vector Core (VVC)P30CA014195 · NCI · SALK INSTITUTE FOR BIOLOGICAL STUDIES · PI Reuben Shaw · 1985 to 2026
$82.8M
Virology CoreP30AI028697 · NIAID · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI KITCHEN, SCOTT G · 1991 to 2018
$39.9M
Lentiviral Vectors to Model Human Gliomas in MiceR01HL053670 · NHLBI · SALK INSTITUTE FOR BIOLOGICAL STUDIES · PI VERMA, INDER MOHAN · 1994 to 2014
$7.5M
REGULATION OF NF-KAPPA BR37AI048034 · NIAID · SALK INSTITUTE FOR BIOLOGICAL STUDIES · PI VERMA, INDER MOHAN · 2000 to 2009
$7.3M
Salk Institute for Biological Studies NINDS Center Core GrantP30NS072031 · NINDS · SALK INSTITUTE FOR BIOLOGICAL STUDIES · PI O'LEARY, DENNIS D · 2011 to 2015
$4.2M
Regulation of NF-Kappa BR01AI048034 · NIAID · SALK INSTITUTE FOR BIOLOGICAL STUDIES · PI VERMA, INDER MOHAN · 2010 to 2014
$3.6M
NCI NIH HHS P30 CA014195NCI NIH HHS P30 CA014195-40NCI NIH HHS P30 CA016042NCI NIH HHS P30 CA14195-38NHLBI NIH HHS HL053670NHLBI NIH HHS R01 HL053670NIAID NIH HHS 5P30 AI028697NIAID NIH HHS AI048034NIAID NIH HHS P30 AI028697NIAID NIH HHS R01 AI048034NINDS NIH HHS P30 NS072031NINDS NIH HHS P30 NS072031-03
6 · The paper itself

Abstract

X-linked Severe Combined Immunodeficiency (SCID-X1) is a genetic disease that leaves newborns at high risk of serious infection and a predicted life span of less than 1 year in the absence of a matched bone marrow donor. The disease pathogenesis is due to mutations in the gene encoding the Interleukin-2 receptor gamma chain (IL-2Rγ), leading to a lack of functional lymphocytes. With the leukemogenic concerns of viral gene therapy there is a need to explore alternative therapeutic options. We have utilized induced pluripotent stem cell (iPSC) technology and genome editing mediated by TALENs to generate isogenic subject-specific mutant and gene-corrected iPSC lines. While the subject-derived mutant iPSCs have the capacity to generate hematopoietic precursors and myeloid cells, only wild-type and gene-corrected iPSCs can additionally generate mature NK cells and T cell precursors expressing the correctly spliced IL-2Rγ. This study highlights the potential for the development of autologous cell therapy for SCID-X1 subjects.

Indexed as

Immunotherapy, AdoptiveRegenerationRegenerative MedicineAntigens, CDBacterial ProteinsCell DifferentiationCell LineDNA RepairDNA Repair EnzymesGenetic TherapyHumansInduced Pluripotent Stem CellsInfantInterleukin Receptor Common gamma SubunitKiller Cells, NaturalMutationAntigens, CDBacterial ProteinsDNA Repair EnzymesInterleukin Receptor Common gamma Subunit

Identifiers

PMID25772073
PMCPMC4545662
OpenAlexW2079995359

What OpenQuestion holds

Textmetadata
LicenceTDM
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 OpenQuestion graph.