Evidence map›Paper›PMID 32483127›Full record

ArticleNature communications2020

Improving the safety of human pluripotent stem cell therapies using genome-edited orthogonal safeguards.

Renata M Martin, Jonas L Fowler, M Kyle Cromer, Benjamin J Lesch, Ezequiel Ponce, Nobuko Uchida, Toshinobu Nishimura, Matthew H Porteus, Kyle M Loh

Open access · goldAbstract read
In one paragraph

Article in Nature communications, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 67 papers.

0numbers the graph read from it
0cells of the map it votes in
67citing papers in PubMed
5.0field-weighted citation impact, top 4% 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

67 citing papers in PubMed, 107 citations in OpenAlex.

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  9. The Role of Gene Therapy and RNA-Based Therapeutic Strategies in Diabetes.International journal of molecular sciences · 2025
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7 more citing papers are in PubMed but not listed here.

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

9 authors at 4 institutions in 2 countries.

Renata M Martin *Institute for Stem Cell Biology & Regenerative Medicine, Stanford University School of Medicine, Stanford, CA, 94305, USA.ORCID http://orcid.org/0000-0003-2996-4260
Jonas L Fowler *Institute for Stem Cell Biology & Regenerative Medicine, Stanford University School of Medicine, Stanford, CA, 94305, USA.
M Kyle CromerInstitute for Stem Cell Biology & Regenerative Medicine, Stanford University School of Medicine, Stanford, CA, 94305, USA.
Benjamin J LeschInstitute for Stem Cell Biology & Regenerative Medicine, Stanford University School of Medicine, Stanford, CA, 94305, USA.
Ezequiel PonceInstitute for Stem Cell Biology & Regenerative Medicine, Stanford University School of Medicine, Stanford, CA, 94305, USA.
Nobuko UchidaInstitute for Stem Cell Biology & Regenerative Medicine, Stanford University School of Medicine, Stanford, CA, 94305, USA.
Toshinobu NishimuraInstitute for Stem Cell Biology & Regenerative Medicine, Stanford University School of Medicine, Stanford, CA, 94305, USA.ORCID http://orcid.org/0000-0002-5425-1708
Matthew H PorteusInstitute for Stem Cell Biology & Regenerative Medicine, Stanford University School of Medicine, Stanford, CA, 94305, USA. mporteus@stanford.edu.ORCID http://orcid.org/0000-0002-3850-4648
Kyle M LohInstitute for Stem Cell Biology & Regenerative Medicine, Stanford University School of Medicine, Stanford, CA, 94305, USA. kyleloh@stanford.edu.ORCID http://orcid.org/0000-0002-8042-0149
Stanford University · USCalifornia Institute for Regenerative Medicine · USInstitute for Stem Cell Biology and Regenerative Medicine · INSilicon Valley University · US

Funding

Graduate Training in Stem Cell Biology and Regenerative MedicineT32GM119995 · NIGMS · STANFORD UNIVERSITY · PI WEISSMAN, IRVING L. · 2017 to 2021
$2.0M
Developing approaches for universal organ transplantationDP5OD024558 · OD · STANFORD UNIVERSITY · PI LOH, KYLE M · 2017 to 2021
$2.0M
NIGMS NIH HHS T32 GM119995NIH HHS DP5 OD024558
6 · The paper itself

Abstract

Despite their rapidly-expanding therapeutic potential, human pluripotent stem cell (hPSC)-derived cell therapies continue to have serious safety risks. Transplantation of hPSC-derived cell populations into preclinical models has generated teratomas (tumors arising from undifferentiated hPSCs), unwanted tissues, and other types of adverse events. Mitigating these risks is important to increase the safety of such therapies. Here we use genome editing to engineer a general platform to improve the safety of future hPSC-derived cell transplantation therapies. Specifically, we develop hPSC lines bearing two drug-inducible safeguards, which have distinct functionalities and address separate safety concerns. In vitro administration of one small molecule depletes undifferentiated hPSCs >10

Indexed as

AnimalsCell Culture TechniquesCell DifferentiationCell SurvivalGene EditingGreen Fluorescent ProteinsHumansMice, Inbred NODMice, KnockoutMice, SCIDNanog Homeobox ProteinPluripotent Stem CellsSmall Molecule LibrariesStem Cell TransplantationTacrolimusTeratomaAP20187Green Fluorescent ProteinsNanog Homeobox ProteinSmall Molecule LibrariesTacrolimus

Identifiers

PMID32483127
PMCPMC7264334
OpenAlexW3031931804

What OpenQuestion 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 OpenQuestion graph.