Disease Focus: Intestinal Disease
hPSC-derived enteric ganglioids for cell therapy in gastrointestinal motility disorders
Research Objective The proposed aims will enable the generation, purification and characterization of enteric neurons from diverse hiPSCs and assessment of their efficacy for cell therapy in GI motility disorders. Impact This proposal addresses a significant unmet clinical need for a cell therapy approach for gastrointestinal motility disorders such as Hirschsprung disease, achalasia and gastroparesis. […]
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 motility disorders including chronic constipation, achalasia and gastroparesis. Major Proposed Activities Assessment of candidate compounds on stem cell derived models from diverse genetic backgrounds Evaluation […]
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 in intestinal fibrosis research is the difficulty in obtaining patient-specific biologically relevant cells for in vitro modeling. This iPSC-derived tool would overcome it. Major Proposed […]
The First Orally Delivered Cell Therapy for the Treatment of Inflammatory Bowel Disease
Research Objective The goal of this project is to develop the first ORAL cell therapy as a breakthrough treatment for inflammatory bowel disease [IBD]. Impact We engineered a new way to deliver cells ORALLY instead of by injection. In doing so, we will better reach the inflamed tissues and provide a much-needed new treatment for […]
ASCENT- Advanced Stem Cell Enteric Neuropathy Therapy
Translational Candidate ASCENT – Advanced Superdonor Cellular Enteric Neuropathy Therapy, is a donor progenitor cell population that replaces the enteric nervous system. Area of Impact ASCENT would treat enteric neuropathies including Hirschsprung disease and total intestinal aganglionosis which currently have no direct therapy Mechanism of Action Our goal is to develop an allogeneic “off the […]
Skin-derived precursor cells for the treatment of enteric neuromuscular dysfunction
The intestine performs the essential function of absorbing food and water into the body. Without a functional intestine, children and adults cannot eat normal meals, and these patients depend on intravenous nutrition to sustain life. Many of these patients do not have a neural system that coordinates the function of the intestine. These patients have […]
Skin-derived precursor cells for the treatment of enteric neuromuscular dysfunction
The intestine performs the essential function of absorbing food and water into the body. Without a functional intestine, children and adults cannot eat normal meals, and these patients depend on intravenous nutrition to sustain life. Many of these patients do not have a neural system that coordinates the function of the intestine. These patients have […]
Stem cell therapy for inflammatory bowel disease
One of the most promising approaches that physicians foresee for treating human disease is regenerative medicine. A major aim in this field is to restore function by repairing damaged organs. Inflammatory bowel disease (IBD) is a chronic disease characterized by intermittent episodes of intestinal inflammation and disruption of the intestinal epithelial barrier. It causes significant […]
The generation and expansion of tissue-engineered small intestine from human stem/ progenitor cells: a preclinical study of functional translation
This proposal aims to complete the preclinical steps to develop tissue-engineered intestine (TESI) as a functional replacement of the small intestine to treat short bowel syndrome (SBS). Common birth conditions especially those associated with prematurity result in SBS wherein 50-75% of the small intestine is gone. SBS children cannot get adequate nutrition and supportive medical […]
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 […]