Disease Focus: Liver Disease
An in vitro and in vivo comparison among three different human hepatic stem cell populations.
Because there is still considerable morbidity and mortality associated with the process of transplantation, and because more than a thousand people die each year while on the liver transplantation list, it is evident that improved and safer liver transplantation would be valuable, as would approaches that provide for an increased number of transplantations in a […]
Assessing the Functional, Immunologic and Microbiologic Characteristics of Human Livers Created in Chimeric Pigs
Research Objective Generating human liver using pig as a bioreactor Impact The livers produced will be used for transplantation into patients with end-stage liver disease, metabolic disorders, and for metastatic liver disease not amenable to resection Major Proposed Activities MILESTONE 1 TASKS 1) Procure liver from control pig 2) Place on XVIVO Perfusion Device MILESTONE […]
iPSCs as a screening tool to predict risk of nonalcoholic fatty liver disease
Research Objective The objective of this proposal is to established undifferentiated iPSCs as a diagnostic tool for the prediction of nonalcoholic fatty liver disease onset and severity. Impact Despite the widespread estimated prevalence of NAFLD, there are currently no tools available to predict likelihood of NAFLD susceptibility beyond standard clinical and demographic information. Major Proposed […]
Universal Pluripotent Liver Failure Therapy (UPLiFT)
Research Objective Universal Pluripotent Liver Failure Therapy (UPLiFT) is composed of 2 lines- UPLiFT0 ( from LiPSC-GR1.1) and UPLiFT1 which will be derived from gene edited universal human pluripotent stem cells. Impact In some liver-based metabolic diseases, replacement of 5-10% of the liver mass may salvage the patient. Transplantation of hepatic progenitors from universal donor […]
Towards hepatocyte cell replacement therapy: developing a renewable source of human hepatocytes from pluripotent stem cells
Research Objective To develop a consistent and abundant source of transplantable human hepatocytes for transplantation. Impact Developing an abundant and consistent source of human hepatocytes that can be used to treat patients with liver failure. Major Proposed Activities To determine the degree by which human pluripotent stem cell (hPSC)-derived hepatocytes engraft and restore liver function […]
Preclinical development of AAV vector-mediated in vivo hepatic reprogramming of myofibroblasts as a therapy for liver fibrosis
Research Objective An intravenously injectable virus that converts the scar cells responsible for liver cirrhosis into the cells that provide most of the liver’s function, thereby preventing or reversing liver failure. Impact The proposed research will develop a new therapy for liver cirrhosis, which can be cured by liver transplantation, but there are not enough […]
Preclinical development of human hepatocyte progenitor cells for cell therapy
Research Objective Determine if human hepatocyte progenitor cells, which exist in the normal adult liver, can be maintained and expanded in vitro while maintaining in vivo regenerative capacity. Impact Cell transplantation therapy can be an effective alternative treatment for severe liver diseases to liver transplantation, which is severely limited by the lack of available donor […]
Generation of bile duct-competent transplantable human liver organoids
Research Objective Generation of human stem cell-derived mini livers capable of exporting bile into the gallbladder after transplantation into the liver Impact Mini livers capable of normal bile export would have potential for therapy of diseases in which bile export is impaired like Alagille syndrome Major Proposed Activities Generation of mini livers using human stem […]
Toshio Miki, USC – CIRM Stem Cell #SciencePitch
Produced by the University of Southern California