Therapeutic potential of three-dimensional hepatocyte-like cells from human adipose-derived mesenchymal stem cells in urea cycle disorders.

Evidence from laboratory and animal studies provides groundwork for understanding mechanisms and potential before human studies continue.

• Level A · Stronger Clinical Evidence
• Level B · Emerging clinical evidence with positive signals
• Level C · Early human research exploring benefits
• Level D · Scientific groundwork from lab and animal studies
• Emerging · Emerging topic under active research

Abstract (original English)

Three-dimensional hepatocyte-like cells (3D HLCs) derived from human adipose-derived mesenchymal stem cells (ADSCs) represent a scalable and clinically accessible cell source for metabolic liver diseases. Here, the authors demonstrate that transplantation (Tx) of ADSC-derived 3D HLCs significantly enhances ammonia detoxification in vivo across multiple urea cycle disorder (UCD) models. 3D HLC Tx attenuated hyperammonemia, accelerated ammonia clearance, and improved encephalopathy-associated behavior in wild-type, ornithine transcarbamylase-deficient, and citrin-deficient mice. Engrafted human cells persisted and contributed to metabolic improvement. These findings highlight ADSC-derived 3D HLCs as a promising, minimally invasive therapeutic strategy and support their potential as an alternative to liver Tx for UCDs.

Evidence level

Evidence from laboratory and animal studies provides groundwork for understanding mechanisms and potential before human studies continue.

Browse all related research

Filter the research library by this study's title keywords, author, or publication year.