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UnknownA hepatitis B virus transgenic mouse model with a conditional, recombinant, episomal genome.

Background & aims

Development of new and more effective therapies against hepatitis B virus (HBV) is limited by the lack of suitable small animal models. The HBV transgenic mouse model containing an integrated overlength 1.3-mer construct has yielded crucial insights, but this model unfortun...
S-EPMC7940981 | biostudies-literature
UnknownHBsAg-redirected T cells exhibit antiviral activity in HBV-infected human liver chimeric mice.

Background

Chronic hepatitis B virus (HBV) infection remains incurable. Although HBsAg-specific chimeric antigen receptor (HBsAg-CAR) T cells have been generated, they have not been tested in animal models with authentic HBV infection.

Methods

We generated a novel CAR targeting HBsAg ...
S-EPMC6038120 | biostudies-literature
UnknownHigh-potency MyoAAV capsids enhanced skeletal muscle correction in a mouse model of GSD IIIa.
Glycogen storage disease type IIIa (GSD IIIa) affects multiple tissues, including liver, heart, and skeletal muscles. We recently reported that an adeno-associated virus serotype 9 vector expressing pullulanase, a bacterial glycogen debranching enzyme, driven by an immunotolerizing dual promoter (AA...
S-EPMC12419012 | biostudies-literature
UnknownA novel humanized mouse lacking murine P450 oxidoreductase for studying human drug metabolism.
Only one out of 10 drugs in development passes clinical trials. Many fail because experimental animal models poorly predict human xenobiotic metabolism. Human liver chimeric mice are a step forward in this regard, as the human hepatocytes in chimeric livers generate human metabolites, but the remain...
S-EPMC5489481 | biostudies-literature
UnknownPCR detection of virulence genes in Yersinia enterocolitica and Yersinia pseudotuberculosis and investigation of virulence gene distribution.
PCR-based assays were developed for the detection of plasmid- and chromosome-borne virulence genes in Yersinia enterocolitica and Yersinia pseudotuberculosis, to investigate the distribution of these genes in isolates from various sources. The results of PCR genotyping, based on 5 virulence-associat...
S-EPMC150046 | biostudies-literature
UnknownCircadian dysfunction induces NAFLD-related human liver cancer in a mouse model.

Background & aims

Chronic circadian dysfunction increases the risk of non-alcoholic fatty liver disease (NAFLD)-related hepatocellular carcinoma (HCC), but the underlying mechanisms and direct relevance to human HCC have not been established. In this study, we aimed to determine whether chro...
S-EPMC10929560 | biostudies-literature
UnknownReprogramming metabolic pathways in vivo with CRISPR/Cas9 genome editing to treat hereditary tyrosinaemia.
Many metabolic liver disorders are refractory to drug therapy and require orthotopic liver transplantation. Here we demonstrate a new strategy, which we call metabolic pathway reprogramming, to treat hereditary tyrosinaemia type I in mice; rather than edit the disease-causing gene, we delete a gene ...
S-EPMC5013601 | biostudies-literature
UnknownCRISPR/Cas9 gene therapy increases the risk of tumorigenesis in the mouse model of hereditary tyrosinemia type I.

Background & aims

The therapeutic potential of CRISPR gene editing has been demonstrated in various animal models; however, little is known about its long-term consequences. This study seeks to bridge this gap by investigating the lasting consequences of CRISPR gene therapy in an animal mode...
S-EPMC11985117 | biostudies-literature
UnknownA human liver chimeric mouse model for non-alcoholic fatty liver disease.

Background & aims

The accumulation of neutral lipids within hepatocytes underlies non-alcoholic fatty liver disease (NAFLD), which affects a quarter of the world's population and is associated with hepatitis, cirrhosis, and hepatocellular carcinoma. Despite insights gained from both human an...
S-EPMC8138774 | biostudies-literature
UnknownA humanized mouse model for adeno-associated viral gene therapy.
Clinical translation of AAV-mediated gene therapy requires preclinical development across different experimental models, often confounded by variable transduction efficiency. Here, we describe a human liver chimeric transgene-free Il2rg-/-/Rag2-/-/Fah-/-/Aavr-/-...
S-EPMC10912671 | biostudies-literature
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