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…
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…
Genome editing of human Tregs to enable combinational tolerogenic therapy with T cell-targeted biologics for T1D
Research Objective Genome-edited autologous regulatory T cells for promoting immune tolerance in combination with T-cell-targeting therapies Impact We focus on type 1 diabetes in this proof-of-concept study, but the therapeutic…
Drug discovery for gastrointestinal motility disorders using hPSC-derived enteric ganglioids
Research Objective Our goal is to use stem cell models to identify a small molecule drug candidate for GI motility disorders. Impact The candidate will be used for severe gastrointestinal…
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…
Gene Therapy for SLC6A8 Creatine Transporter Disorder
Research Objective The objective is to define a final therapeutic candidate for an effective gene therapy for mutations of the creatine transporter SLC6A8, a major cause of X-linked intellectual disability…
A Novel Therapy for Sanfilippo B
Research Objective To develop a Stem Cell therapy for Sanfilippo B syndrome. Impact There is no treatment for Sanfilippo syndrome, and other therapeutic approaches have failed in clinics. This proposal…
Bioengineering human stem cell-derived beta cell organoids to monitor cell health in real time and improve therapeutic outcomes in patients
Research Objective We will generate nanoprobe-containing stem cell-derived human beta cells that can be monitored in real time in response to inflammatory stress upon transplantation in patients with type 1…
A new precision medicine based iPSC-derived model to study personalized intestinal fibrosis treatments in pediatric patients with Crohn’s disease
Research Objective We propose to discover a tool that will utilize patient specific iPSC-derived human mini-guts to identify personalized antifibrotic treatments in pediatric Crohn’s disease patients Impact The major bottleneck…
Extending Immune-Evasive Human Islet-Like Organoids (HILOs) Survival and Function as a Cure for T1D
Research Objective Determine optimal islet transplant conditions and systemic treatments that promote graft survival upon transplantation into immune-competent diabetic subjects. Impact Our proposal will optimize the generation and viability of…
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