Disease Focus: Metabolic Disorders


Pluripotent and Somatic Stem Cell Models to Study Inherited Diarrheal Disorders

Our research group at [REDACTED] has had a long-standing interest in understanding the cause of several disorders that result in severe, and often times fatal forms of diarrhea in children. These diarrheal disorders are inherited, and somehow lead to poor absorption of nearly all forms of nutrients, including protein, sugars and fats. Why children with […]

Liver Cell Transplantation

Because there is still considerable morbidity and mortality associated with the process of whole liver transplantation, and because more than a thousand people die each year while on the liver transplantation list, and tens of thousands more never get on the list because of the lack of available livers, it is evident that improved and […]

Methods for detection and elimination of residual human embryonic stem cells in a differentiated cell product

Human embryonic stem cells (hESC), and other related pluripotent stem cells, have great potential as starting material for the manufacture of curative cell therapies. This is primarily for two reasons. First, by manipulating cues in their cell culture conditions, these cells can be directed to become essentially any desired human cell type (a property known […]

Developing induced pluripotent stem cells into human therapeutics and disease models

Human embryonic stem cells (hESCs) can undergo unlimited self-renewal and differentiate into all the cell types in the human body, and thus hold great promise for cell replacement therapy. However, one major problem for hESC-based therapy is that the cells derived from hESCs will be rejected by the recipient and can only be tolerated under […]

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 […]