Evidence map›Paper›PMID 27500178›Full record

ArticleMolecular therapy. Methods & clinical development2016

Investigating the functionality of an OCT4-short response element in human induced pluripotent stem cells.

Agustin Vega-Crespo, Brian Truong, Kip J Hermann, Jason P Awe, Katherine M Chang, Patrick C Lee, Benjamen E Schoenberg, Lily Wu, James A Byrne, Gerald S Lipshutz

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

Article in Molecular therapy. Methods & clinical development, 2016. 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, top 95% 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

2 citing papers in PubMed, 2 citations in OpenAlex.

  1. Article
  2. Article
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

10 authors at 3 institutions in 1 country.

Agustin Vega-CrespoDepartment of Molecular and Medical Pharmacology, Center for Health Sciences, University of California, Los Angeles, alifornia, USA; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, University of California, Los Angeles, alifornia, USA.
Brian TruongDepartment of Molecular and Medical Pharmacology, Center for Health Sciences, University of California, Los Angeles, alifornia, USA; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, University of California, Los Angeles, alifornia, USA.
Kip J HermannDepartment of Molecular and Medical Pharmacology, Center for Health Sciences, University of California , Los Angeles, alifornia, USA.
Jason P AweDepartment of Molecular and Medical Pharmacology, Center for Health Sciences, University of California, Los Angeles, alifornia, USA; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, University of California, Los Angeles, alifornia, USA.
Katherine M ChangDepartment of Molecular and Medical Pharmacology, Center for Health Sciences, University of California, Los Angeles, alifornia, USA; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, University of California, Los Angeles, alifornia, USA.
Patrick C LeeDepartment of Molecular and Medical Pharmacology, Center for Health Sciences, University of California, Los Angeles, alifornia, USA; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, University of California, Los Angeles, alifornia, USA.
Benjamen E SchoenbergDepartment of Molecular and Medical Pharmacology, Center for Health Sciences, University of California, Los Angeles, alifornia, USA; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, University of California, Los Angeles, alifornia, USA.
Lily WuDepartment of Molecular and Medical Pharmacology, Center for Health Sciences, University of California , Los Angeles, alifornia, USA.
James A ByrneDepartment of Molecular and Medical Pharmacology, Center for Health Sciences, University of California, Los Angeles, alifornia, USA; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, University of California, Los Angeles, alifornia, USA.
Gerald S LipshutzDepartment of Molecular and Medical Pharmacology, Center for Health Sciences, University of California, Los Angeles, alifornia, USA; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, University of California, Los Angeles, alifornia, USA; Department of Surgery, UCLA, Los Angeles, California, USA; Department of Medicine, UCLA, Los Angeles, California USA; Department of Psychiatry, Los Angeles, California USA; Department of Urology, UCLA, Los Angeles, California USA; The Intellectual and Developmental Disabilities Research Center at UCLA, Los Angeles, California USA; The Semel Institute for Neuroscience, Los Angeles, California USA.
Oklahoma State University Center for Health Sciences · USBroad Center · USUniversity of California, Los Angeles · US

Funding

UCLA MSD ProgramR25GM055052 · NIGMS · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI BARBER, PAUL HENRY, HASSON, TAMA W. · 1996 to 2021
$9.2M
NIGMS NIH HHS R25 GM055052
6 · The paper itself

Abstract

Pluripotent stem cells offer great therapeutic promise for personalized treatment platforms for numerous injuries, disorders, and diseases. Octamer-binding transcription factor 4 (OCT4) is a key regulatory gene maintaining pluripotency and self-renewal of mammalian cells. With site-specific integration for gene correction in cellular therapeutics, use of the OCT4 promoter may have advantages when expressing a suicide gene if pluripotency remains. However, the human OCT4 promoter region is 4 kb in size, limiting the capacity of therapeutic genes and other regulatory components for viral vectors, and decreasing the efficiency of homologous recombination. The purpose of this investigation was to characterize the functionality of a novel 967bp OCT4-short response element during pluripotency and to examine the OCT4 titer-dependent response during differentiation to human derivatives not expressing OCT4. Our findings demonstrate that the OCT4-short response element is active in pluripotency and this activity is in high correlation with transgene expression in vitro, and the OCT4-short response element is inactivated when pluripotent cells differentiate. These studies demonstrate that this shortened OCT4 regulatory element is functional and may be useful as part of an optimized safety component in a site-specific gene transferring system that could be used as an efficient and clinically applicable safety platform for gene transfer in cellular therapeutics.

Identifiers

PMID27500178
PMCPMC4954563
OpenAlexW2493481998

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.