Evidence mapPaperPMID 35803227Full record

ArticleCell stem cell2022

A scalable organoid model of human autosomal dominant polycystic kidney disease for disease mechanism and drug discovery.

Tracy Tran, Cheng Jack Song, Trang Nguyen, Shun-Yang Cheng, Jill A McMahon, Rui Yang, Qiuyu Guo, Balint Der, Nils O Lindström, Daniel C-H Lin and 1 more

Open access · greenAbstract read
In one paragraph

Article in Cell stem cell, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 83 papers.

0numbers the graph read from it
0cells of the map it votes in
83citing papers in PubMed
10.0field-weighted citation impact, top 1% 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

83 citing papers in PubMed, 124 citations in OpenAlex.

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23 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

11 authors at 2 institutions in 1 country.

Tracy TranDepartment of Stem Cell Biology and Regenerative Medicine, Broad-CIRM Center, Keck School of Medicine, University of Southern California, Los Angeles, CA 90089, USA.
Cheng Jack SongDepartment of Stem Cell Biology and Regenerative Medicine, Broad-CIRM Center, Keck School of Medicine, University of Southern California, Los Angeles, CA 90089, USA; Amgen Research, Cardiometabolic Disorders, 1120 Veterans Blvd, South San Francisco, CA 94080, USA.
Trang NguyenDepartment of Stem Cell Biology and Regenerative Medicine, Broad-CIRM Center, Keck School of Medicine, University of Southern California, Los Angeles, CA 90089, USA.
Shun-Yang ChengDepartment of Stem Cell Biology and Regenerative Medicine, Broad-CIRM Center, Keck School of Medicine, University of Southern California, Los Angeles, CA 90089, USA.
Jill A McMahonDepartment of Stem Cell Biology and Regenerative Medicine, Broad-CIRM Center, Keck School of Medicine, University of Southern California, Los Angeles, CA 90089, USA.
Rui YangDepartment of Stem Cell Biology and Regenerative Medicine, Broad-CIRM Center, Keck School of Medicine, University of Southern California, Los Angeles, CA 90089, USA.
Qiuyu GuoDepartment of Stem Cell Biology and Regenerative Medicine, Broad-CIRM Center, Keck School of Medicine, University of Southern California, Los Angeles, CA 90089, USA.
Balint DerDepartment of Stem Cell Biology and Regenerative Medicine, Broad-CIRM Center, Keck School of Medicine, University of Southern California, Los Angeles, CA 90089, USA.
Nils O LindströmDepartment of Stem Cell Biology and Regenerative Medicine, Broad-CIRM Center, Keck School of Medicine, University of Southern California, Los Angeles, CA 90089, USA.
Daniel C-H LinAmgen Research, Cardiometabolic Disorders, 1120 Veterans Blvd, South San Francisco, CA 94080, USA.
Andrew P McMahonDepartment of Stem Cell Biology and Regenerative Medicine, Broad-CIRM Center, Keck School of Medicine, University of Southern California, Los Angeles, CA 90089, USA. Electronic address: amcmahon@med.usc.edu.
University of Southern California · USAmgen (United States) · US

Funding

CELL INTERACTIONS IN DEVELOPMENT OF THE MAMMALIAN KIDNEYR01DK054364 · NIDDK · UNIVERSITY OF SOUTHERN CALIFORNIA · PI ANDREW P. MCMAHON · 1998 to 2022
$3.7M
Training in Developmental Biology, Stem Cells and RegenerationT32HD060549 · UNIVERSITY OF SOUTHERN CALIFORNIA · 2025 to 2025
$223k
NICHD NIH HHS T32 HD060549NIDDK NIH HHS R01 DK054364NIDDK NIH HHS R37 DK054364
6 · The paper itself

Abstract

Human pluripotent stem-cell-derived organoids are models for human development and disease. We report a modified human kidney organoid system that generates thousands of similar organoids, each consisting of 1-2 nephron-like structures. Single-cell transcriptomic profiling and immunofluorescence validation highlighted patterned nephron-like structures utilizing similar pathways, with distinct morphogenesis, to human nephrogenesis. To examine this platform for therapeutic screening, the polycystic kidney disease genes PKD1 and PKD2 were inactivated by gene editing. PKD1 and PKD2 mutant models exhibited efficient and reproducible cyst formation. Cystic outgrowths could be propagated for months to centimeter-sized cysts. To shed new light on cystogenesis, 247 protein kinase inhibitors (PKIs) were screened in a live imaging assay identifying compounds blocking cyst formation but not overall organoid growth. Scaling and further development of the organoid platform will enable a broader capability for kidney disease modeling and high-throughput drug screens.

Indexed as

CystsPolycystic Kidney, Autosomal DominantDrug DiscoveryHumansKidneyOrganoidsTRPP Cation ChannelsTRPP Cation Channelsdevelopmental trajectorydisease modelingdrug screenhigh throughputkidney developmentkidney organoidnephron developmentphenotypic screenpolycystic kidney diseasescRNA-seq

Identifiers

PMID35803227
PMCPMC11088748
OpenAlexW4284704161

What Socratic 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 Socratic graph.