Grant Award Details

hPSC-derived enteric ganglioids for cell therapy in gastrointestinal motility disorders
Grant Type: 
Grant Number: 
DISC0-14521
Project Objective: 
  • To generate hPSC-derived enteric ganglioids that can be transplanted and engraft in the gut of patients with motility disorders and restore motility dysfunction.
Investigator: 
Disease Focus: 
Intestinal Disease
Metabolic Disorders
Neurological Disorders
Other
Human Stem Cell Use: 
iPS Cell
Cell Line Generation: 
iPS Cell
Award Value: 
$1,589,307
Status: 
Pre-Active

Grant Application Details

Application Title: 
  • hPSC-derived enteric ganglioids for cell therapy in gastrointestinal motility disorders
Public Abstract: 

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.

Major Proposed Activities

  • Standardize the generation of enteric neurons from stem cells of diverse backgrounds
  • Developing methods to speed up the process of enteric neuron production from stem cells
  • Evaluating the ability of stem cell derived enteric neurons to survive in the host tissue and rescue the disease in mouse models
Statement of Benefit to California: 

Gastrointestinal (GI) motility disorders are severe and common medical conditions resulting from dysfunction or degeneration of the enteric nervous system (ENS). The ENS is an extensive network of neurons inside the gut tissue that are responsible for local regulation of motility and digestion. This proposal is aimed at developing stem cell based therapies to replace the damaged or absent enteric neurons in motility disorders such as Hirschsprung disease, achalasia and gastroparesis.

  • Share
PrintPDF version