A5025904108- Organ Transplantation Techniques and Outcomes
- Liver physiology and pathology
- Metabolism and Genetic Disorders
- Virus-based gene therapy research
- Viral gastroenteritis research and epidemiology
- Xenotransplantation and immune response
- Liver Disease and Transplantation
- Cancer Mechanisms and Therapy
- Clinical Nutrition and Gastroenterology
- Tissue Engineering and Regenerative Medicine
Mayo Clinic in Arizona
2016-2025
Mayo Clinic
2016-2024
Diminished hepatocyte regeneration is a key feature of acute and chronic liver diseases after extended resections, resulting in the inability to maintain or restore sufficient functional mass. Therapies are lacking, making transplantation only curative option for end-stage disease. Here, we report on structure-based development characterization (nuclear magnetic resonance [NMR] spectroscopy) first-in-class small molecule inhibitors dual-specificity kinase MKK4 (MKK4i). MKK4i increased upon...
Transplantation of gene-corrected autologous hepatocytes can cure metabolic disease in a preclinical pig model hereditary tyrosinemia type 1.
Orthotopic liver transplantation remains the only curative therapy for inborn errors of metabolism. Given tremendous success primary immunodeficiencies using ex-vivo gene with lentiviral vectors, there is great interest in developing similar therapies metabolic diseases. We have previously generated a pig model hereditary tyrosinemia type 1 (HT1), an autosomal recessive disorder caused by deficiency fumarylacetoacetate hydrolase (FAH). Using this model, we demonstrated and cell vector to...