ArticleStem cell research & therapy2025
First-in-human clinical study of an embryonic stem cell product for urea cycle disorders.
Article in Stem cell research & therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.
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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.
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.
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Who cites it
6 citing papers in PubMed.
- In-utero cell transplantation for hypophosphatasia with gene-edited hESC-derived MSCs in a murine model.Molecular therapy. Advances · 2026Article
- The feasibility of human embryonic stem cell (hESC)-derived cartilage for tracheoplasty.Regenerative therapy · 2025Article
- Generation of implant-type tissue-engineered cartilage from human embryonic stem cell-derived chondrocytes.Regenerative therapy · 2025Article
- Stem cells as biological drugs for incurable diseases.Stem cell research & therapy · 2025Article
- From Adipose to Action: Reprogramming Stem Cells for Functional Neural Progenitors for Neural Regenerative Therapy.International journal of molecular sciences · 2025Review
- Primitive Hepatoblasts Driving Early Liver Development.bioRxiv : the preprint server for biology · 2025Article
Corrections and comments
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Authors and funding
39 authors.
Funding
Abstract
backgroundThis study assesses the safety and efficacy of hepatocyte-like cell (HLC) infusion therapy derived from human embryonic stem cells as bridging therapy for neonatal-onset urea cycle disorders (UCD). The research includes both preclinical and clinical evaluations to determine the feasibility of HLC infusion as a therapeutic option for safer pediatric liver transplantation.
methodsPreclinical studies were conducted to validate the safety, biodistribution, and ammonia metabolism capabilities of HLCs using SCID mice models of UCD and extensive animal studies. In the clinical trial, five neonates with UCD received HLC infusions, intending to maintain metabolic stability and exceed a target weight of over 6 kg, which is considered necessary for safer liver transplantation.
resultsPreclinical studies demonstrated that HLCs successfully engrafted in the liver without adverse migration or tumor formation and effectively elongated survival. Clinically, all five neonates exceeded the target weight of 6 kg while maintaining metabolic stability and successfully bridging to transplantation. Post-transplantation follow-up revealed stable growth, metabolic control, and no neurological complications.
conclusionsThe combined preclinical and clinical findings support HLC infusion as a viable bridge therapy for neonates with UCD, providing metabolic support to achieve safer weight thresholds for transplantation. While promising, careful monitoring remains essential, particularly for potential complications such as thrombus formation.
trial registrationjRCT, jRCT1090220412. Registered on 27 February 2019, https://jrct.niph.go.jp/en-latest-detail/jRCT1090220412 (originally registered in JMACCT (JMA-IIA00412)).
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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.