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Liver fibrosis negatively impacts in vivo gene transfer to murine hepatocytes.
Nat Commun
March 2025
San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Liver fibrosis occurs in several genetic and acquired disease conditions, leading to alterations of the tissue and metabolism, which may adversely affect viral vector-mediated gene therapy. Here, we assessed the impact of liver fibrosis on in vivo gene transfer to hepatocytes mediated by lentiviral vectors or adeno-associated viral vectors. We exploited two chemically induced fibrosis mouse models characterized by tissue damage in different areas of the liver lobule.
View Article and Find Full Text PDFOral Cornstarch and Glycyrrhizin Improve Severe Liver Injury Caused by Glycogen Storage Disease Type IXa.
Cureus
February 2025
Division of Pediatrics and Perinatology, Faculty of Medicine, Tottori University, Tottori, JPN.
A previously healthy 18-month-old boy presented with hepatomegaly, accompanied by liver injury. Imaging and liver biopsy findings suggested a hepatic glycogen storage disease (GSD) but not GSD type I. Genetic testing revealed a partial deletion of the PHKA2 gene, confirming the diagnosis of GSD type IXa.
View Article and Find Full Text PDFRecurrence of Myopathy After Liver Transplantation for Patients With End-Stage GSD Type IIIa.
Transplant Proc
March 2025
Department of Critical Liver Diseases, Liver Research Center, Beijing Friendship Hospital, Capital Medical University, Beijing, China; Liver Transplantation Center, National Clinical Research Center for Digestive Diseases. Beijing Friendship Hospital, Capital Medical University, Beijing, China; Pediatric Liver Transplantation Research Center, Capital Medical University, Beijing, China. Electronic address:
Background: Glycogen storage disease (GSD) is an inherited metabolic disorder affecting glycogen metabolism. The overall incidence of GSD is estimated to be 1 in every 20,000 to 43,000 newborns. GSD is classified into 12 types based on the enzyme deficiency and the tissues affected.
View Article and Find Full Text PDFA comparative genomic study across 396 liver biopsies provides deep insight into FGF21 mode of action as a therapeutic agent in metabolic dysfunction-associated steatotic liver disease.
Clin Transl Med
February 2025
Centre for Pharmacology and Toxicology, Hannover Medical School, Hannover, Germany.
Background: Metabolic dysfunction-associated steatotic liver disease (MASLD) is a systemic disease with insulin resistance at its core. It affects one-third of the world population. Fibroblast growth factor (FGF21)-based therapies are effective in lowering hepatic fat content and fibrosis resolution; yet, its molecular functions remain uncertain.
View Article and Find Full Text PDFGlycogen storage disease type III (GSD III) is a rare metabolic disorder characterized by a deficiency of liver and muscle amylo-1,6-glucosidase. This condition presents with severe hepatic symptoms in childhood, mostly hepatomegaly, hypoglycemia in half of patients, while muscular complications may predominate in adulthood. Hepatic fibrosis, cirrhosis and hepatocellular carcinoma (HCC) are common complications in older patients.
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