Disease Focus: Metabolic Disorders


Stem cell tolerance through the use of engineered antigen-specific regulatory T cells

Type 1 Diabetes (T1D) occurs as a consequence of uncontrolled immune activation, culminating in the destruction of insulin-producing beta-cells. Efforts to prevent or reverse diabetes have been limited by the lack of safe and effective immunotherapies coupled with the inability to restore insulin producing beta-cells. We believe proper immune control to self-tissues to be a […]

Development of the Theracyte Cellular Encapsulation System for Delivery of human ES Cell-derived Pancreatic Islets and Progenitors.

There are several challenges to the successful implementation of a cellular therapy for insulin dependent diabetes derived from Human Embryonic Stem Cells (hESCs). Among these are the development of functional insulin-producing cells, a clinical delivery method that eliminates the need for chronic immunosuppression, and assurance that hESC-derived tumors do not develop in the patient. We […]

Development of an hES Cell-Based Assay System for Hepatocyte Differentiation Studies and Predictive Toxicology Drug Screening

Drug-induced liver toxicity, including that from FDA-approved drugs, is the leading cause of liver failure in the US. One of the biggest road blocks to testing drug-induced liver toxicity prior to clinical studies or release of the drug into the market is the absence of a good model of human drug metabolism in the liver. […]

Molecular dissection of adult liver regeneration to guide the generation of hepatocytes from pluripotent stem cells

The liver is a promising target for cell therapy since it supports and functionally integrates transplanted cells. Human liver contains more than 50 billion cells and more than 10% replacement will be required for most liver diseases. Hence, embryonic stem cells (ESC), which have unlimited growth capacities, represent one of the few cell types with […]

Mechanism of Tissue Engineered Small Intestine Formation

Short Bowel Syndrome is an expensive, morbid condition with an increasing incidence. Fundamental congenital and perinatal conditions such as gastroschisis, malrotation, atresia, and necrotizing enterocolitis (NEC) may lead to short bowel syndrome (SBS). NEC is the most common gastrointestinal emergency in neonates and primarily occurs in premature infants As rates of prematurity are increasing, so […]

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 […]

Endodermal differentiation of human ES cells

The goals of this proposal are to investigate endodermal differentiation and proliferation in human ES cell cultures. Endodermal cells give rise to the epithelial lining of the respiratory and digestive tract as well as to the liver and pancreas. The future treatment of diseases such as type I diabetes using stem cell therapy relies on […]

Differentiation of Human Embryonic Stem Cells to Intestinal Fates

The roughly 25 feet of intestine in the adult human play numerous essential roles in daily life, such as nutrient absorption, secretion of hormones, and serving as a barrier to infection. Commensurate with these diverse roles, diseases of the intestine are a considerable source of human morbidity and mortality. Indeed, numerous pathologic conditions including inflammatory […]

Cell Therapy for Diabetes

Diabetes exacts a tremendous toll on patients, their families, and society in general. Autoimmune Type 1 diabetes, often called juvenile-onset diabetes, is caused by a person’s own immune system mistakenly destroying their insulin-producing cells in the pancreas, known as beta cells. When those beta cells are lost, the ability to produce insulin in response to […]