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Conversion the Intestinal Smooth Muscle Cells of Hirschsprung’s Disease into Enteric Neurons-like Cells by Small Molecule Compounds
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Read the article on jpedsurg.org ↗Article · Dec 2025 · 1 min read
In brief
In brief
This research explores converting intestinal smooth muscle cells from Hirschsprung's disease patients into enteric neuron-like cells using small molecule compounds, addressing the challenge of obtaining sufficient cells for ENS transplantation therapy. The approach offers a potential non-genetic editing solution for treating this congenital disorder that affects gut function in approximately 1 in 5,000 newborns.
- The enteric nervous system (ENS) is a complex neuronal network in the gut wall essential for gastrointestinal function regulation.
- Hirschsprung's disease results from ENS defects and affects approximately 1 in 5,000 live births worldwide.
- ENS transplantation shows promise as a treatment approach but faces challenges in sourcing adequate non-gene edited ENS tissue.
- Small molecule compounds may enable conversion of intestinal smooth muscle cells into enteric neuron-like cells for therapeutic use.
Written by the GCMD Library team from the article.
The enteric nervous system (ENS), often referred to as the "second brain," is a complex network of neurons and glial cells in the gut wall, crucial for regulating its functions. Defects in the ENS can lead to Hirschsprung’s disease (HSCR), a serious congenital disorder affecting approximately 1 in 5,000 live births. A promising approach is ENS transplantation. However, obtaining sufficient non-gene edited ENS remains a significant challenge.
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