Therapeutic/Technology: Technology
Development of an immune tolerant hESC source for allogeneic cell therapy applications
Human embryonic stem cells (hESCs) are an ideal tissue source for cell replacement therapy (CRT). They have the potential for limitless self-renewal while retaining their ability to differentiate into a wide variety of cells and tissues. Since their first derivation in 1998, hESCs have been used in many studies in order to evaluate their potential […]
Generation of regulatory T cells by reprogramming
The goal of our research is to develop efficient methods for making a particular class of immune-system cells known as regulatory T cells (Tregs). Tregs have the potential to be useful in a wide variety of clinical situations. For instance, they could be used to control the harmful immune responses seen in patients with autoimmune […]
Maternal and Fetal Immune Responses to In Utero Hematopoietic Stem Cell Transplantation
The immune system is the body’s defense system against disease and can recognize foreign cells. Because of this, stem cells and organs that are transplanted from one person to another are usually “rejected” by the immune system, forcing doctors to use powerful immune suppressive drugs with severe side effects. This natural defense system will therefore […]
Thymus based tolerance to stem cell therapies
This proposal focuses on the role of the immune system in transplantation of derivatives of human pluripotent stem cells (hPSCs). A critical roadblock to successful cell replacement therapies, no matter what the disease or injury, is the fact that the immune system’s main function is to prevent the introduction of foreign substances into our bodies. […]
Purified allogeneic hematopoietic stem cells as a platform for tolerance induction
Blood and immune cells originate and mature in the bone marrow. Bone marrow cells are mixtures of blood cells at different stages of development, and include rare populations of blood-forming stem cells. These stem cells are the only cells capable of generating the blood system for the life of an individual. Bone marrow transplants (BMT) […]
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 […]
Stem cell differentiation to thymic epithelium for inducing tolerance to stem cells
The thymus is an organ that plays a key role in controlling immune responses and immune tolerance. The thymus promotes immune tolerance by deleting and removing self-reactive T cells from the immune system. In addition, the thymus also helps drive the production of important suppressor T cell populations like regulatory T cells that also control […]
Generation of disease models for neurodegenerative disorders in hESCs by gene targeting
The ability to target a specific locus in the mouse genome and to alter it in a specific fashion has fundamentally changed experimental design and made mice the preeminent model for studying human diseases . However, pathogenesis in humans have unique pathways that may not be revealed by only using mouse or other animal models. […]
Discovery of adhesion ligands for pluripotent human stem cells
We have assembled a team of investigators with complementary expertise in applying the state-of-the-art “one-bead-one-compound” (OBOC) combinatorial library methods to identify synthetic chemical molecules that bind to unique receptors (protein molecules) on the surface of human embryonic stem cells and induced pluripotent stem cells. In this technology, stem cells will be mixed with huge number […]
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 […]