Evidence map›Paper›PMID 30651323›Full record

ArticleScience translational medicine2019

Clinical-grade stem cell-derived retinal pigment epithelium patch rescues retinal degeneration in rodents and pigs.

Ruchi Sharma, Vladimir Khristov, Aaron Rising, Balendu Shekhar Jha, Roba Dejene, Nathan Hotaling, Yichao Li, Jonathan Stoddard, Casey Stankewicz, Qin Wan and 16 more

Erratum issuedOpen access · greenAbstract read
In one paragraph

Article in Science translational medicine, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 191 papers, 3 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
191citing papers in PubMed, 3 pooled it
14.7field-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

191 citing papers in PubMed, 3 syntheses or guidelines pooled it, 360 citations in OpenAlex.

  1. Pooled it
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  14. Autophagy in ocular diseases: from mechanisms to therapeutic potential.Frontiers in cell and developmental biology · 2026
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131 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

26 authors at 5 institutions in 2 countries.

Ruchi SharmaUnit on Ocular and Stem Cell Translational Research, National Eye Institute, NIH, Bethesda, MD 20892, USA.ORCID 0000-0002-2138-6462
Vladimir KhristovSection on Epithelial and Retinal Physiology and Disease, National Eye Institute, NIH, Bethesda, MD 20892, USA.ORCID 0000-0002-7778-3993
Aaron RisingSection on Epithelial and Retinal Physiology and Disease, National Eye Institute, NIH, Bethesda, MD 20892, USA.ORCID 0000-0002-0725-9758
Balendu Shekhar JhaUnit on Ocular and Stem Cell Translational Research, National Eye Institute, NIH, Bethesda, MD 20892, USA.ORCID 0000-0002-6509-8171
Roba DejeneUnit on Ocular and Stem Cell Translational Research, National Eye Institute, NIH, Bethesda, MD 20892, USA.
Nathan HotalingUnit on Ocular and Stem Cell Translational Research, National Eye Institute, NIH, Bethesda, MD 20892, USA.
Yichao LiVisual Function Core, National Eye Institute, NIH, Bethesda, MD 20892, USA.ORCID 0000-0003-2697-2556
Jonathan StoddardCasey Eye Institute, Oregon Health & Science University, Portland, OR 97239, USA.
Casey StankewiczCellular Dynamics International Inc. (a FUJIFILM company), Madison, WI 53711, USA.
Qin WanSection on Epithelial and Retinal Physiology and Disease, National Eye Institute, NIH, Bethesda, MD 20892, USA.
Connie ZhangSection on Epithelial and Retinal Physiology and Disease, National Eye Institute, NIH, Bethesda, MD 20892, USA.
Mercedes Maria CamposHistology Core, National Eye Institute, NIH, Bethesda, MD 20892, USA.ORCID 0000-0003-1331-1484
Kiyoharu J MiyagishimaSection on Epithelial and Retinal Physiology and Disease, National Eye Institute, NIH, Bethesda, MD 20892, USA.
David McGaugheyOphthalmic Genetics and Visual Functional Branch, National Eye Institute, NIH, Bethesda, MD 20892, USA.ORCID 0000-0002-9224-2888
Rafael VillasmilFlow Cytometry Core, National Eye Institute, NIH, Bethesda, MD 20892, USA.ORCID 0000-0002-8956-2979
Mary MattapallilLaboratory of Immunology, National Eye Institute, NIH, Bethesda, MD 20892, USA.
Boris StanzelMacula Center Saar, Sulzbach Knappschaft Eye Clinic, Sulzbach/Saar 66280, Germany.
Haohua QianVisual Function Core, National Eye Institute, NIH, Bethesda, MD 20892, USA.ORCID 0000-0002-1114-4697
Wai WongUnit on Neuron-Glia Interactions in Retinal Disease, National Eye Institute, NIH, Bethesda, MD 20892, USA.ORCID 0000-0003-0681-4016
Lucas ChaseCellular Dynamics International Inc. (a FUJIFILM company), Madison, WI 53711, USA.
Steve CharlesCharles Retina Institute, Germantown, TN 38138, USA.
Trevor McGillCasey Eye Institute, Oregon Health & Science University, Portland, OR 97239, USA.
Sheldon MillerSection on Epithelial and Retinal Physiology and Disease, National Eye Institute, NIH, Bethesda, MD 20892, USA.ORCID 0000-0001-7495-2547
Arvydas MaminishkisSection on Epithelial and Retinal Physiology and Disease, National Eye Institute, NIH, Bethesda, MD 20892, USA.ORCID 0000-0003-3345-3375
Juan AmaralOffice of Scientific Director, National Eye Institute, NIH, Bethesda, MD 20892, USA.ORCID 0000-0001-8755-9170
Kapil BhartiUnit on Ocular and Stem Cell Translational Research, National Eye Institute, NIH, Bethesda, MD 20892, USA. Kapilbharti@nei.nih.gov.ORCID 0000-0003-4884-3173
National Eye Institute · USCellular Dynamics International (United States) · USOregon Health & Science University · USKnappschaftsklinikum Saar · DERetina Institute · US

Funding

Developing autologous iPSC-RPE based IND for AMD patientsZIAEY000542 · NEI · NATIONAL EYE INSTITUTE · PI BHARTI, KAPIL · 2014 to 2025
$24.2M
Developing patient-specific iPS cell derived RPE disease modelsZIAEY000532 · NEI · NATIONAL EYE INSTITUTE · PI BHARTI, KAPIL · 2013 to 2025
$22.0M
Visual Function CoreZICEY000503 · NEI · NATIONAL EYE INSTITUTE · PI QIAN, HAOHUA · 2011 to 2025
$12.3M
Biological function microRNAs enriched in RPE: in vitro and in vivo modelsZIAEY000456 · NEI · NATIONAL EYE INSTITUTE · PI SCHNEEWEIS, DAVID · 2009 to 2020
$9.5M
Histopathology CoreZICEY000461 · NEI · NATIONAL EYE INSTITUTE · PI SCHNEEWEIS, DAVID · 2009 to 2022
$7.7M
Evaluation of Visual Function in Animal ModelsZIAEY000530 · NEI · NATIONAL EYE INSTITUTE · PI QIAN, HAOHUA · 2013 to 2025
$1.8M
Intramural NIH HHS ZIA EY000532
6 · The paper itself

Abstract

Considerable progress has been made in testing stem cell-derived retinal pigment epithelium (RPE) as a potential therapy for age-related macular degeneration (AMD). However, the recent reports of oncogenic mutations in induced pluripotent stem cells (iPSCs) underlie the need for robust manufacturing and functional validation of clinical-grade iPSC-derived RPE before transplantation. Here, we developed oncogenic mutation-free clinical-grade iPSCs from three AMD patients and differentiated them into clinical-grade iPSC-RPE patches on biodegradable scaffolds. Functional validation of clinical-grade iPSC-RPE patches revealed specific features that distinguished transplantable from nontransplantable patches. Compared to RPE cells in suspension, our biodegradable scaffold approach improved integration and functionality of RPE patches in rats and in a porcine laser-induced RPE injury model that mimics AMD-like eye conditions. Our results suggest that the in vitro and in vivo preclinical functional validation of iPSC-RPE patches developed here might ultimately be useful for evaluation and optimization of autologous iPSC-based therapies.

Indexed as

AnimalsDisease Models, AnimalMacular DegenerationRatsRetinal DegenerationRetinal Pigment EpitheliumStem CellsSwine

Identifiers

PMID30651323
PMCPMC8784963
OpenAlexW2908602993

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.