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Transgenic Mouse Model of Congenital Choledochal Cyst.
Hannah Nicole Rinehardt1, Alexis Martyn1, Alexander Kolodychak1
1Children's Hospital of Pittsburgh.
Research Square
|November 24, 2025
Summary
Researchers developed a new mouse model for congenital choledochal cysts, a rare liver disease. This model mimics human disease, aiding further study of its carcinogenic nature and potential treatments.
Area of Science:
- Hepatobiliary Medicine
- Gastroenterology
- Translational Research
Background:
- Choledochal cysts are rare congenital hepatobiliary tree dilations.
- Complete resection is recommended due to malignancy risk.
- Metachronous cholangiocarcinoma risk necessitates lifelong surveillance post-resection.
Purpose of the Study:
- To develop a reliable mouse model for choledochal cysts.
- To mimic the human disease process, including its connection to carcinogenesis.
- To facilitate further research into choledochal cyst pathophysiology.
Main Methods:
- Experimental transgenic mice (Pdx-Cre, TGFα, LSL-KRAS G12D Mu/Wt) were bred.
- Control C57 mice were used for comparison.
- Serial abdominal magnetic resonance imaging (MRI) was performed from weaning to sacrifice.
Main Results:
- All experimental mice developed fusiform, extrahepatic common bile duct dilation, mimicking Type I choledochal cyst.
- Choledochal cysts were detectable via imaging upon weaning.
- Maximum common bile duct diameter was significantly larger in experimental mice by 10 weeks.
Conclusions:
- A transgenic mouse model successfully replicates congenital choledochal cyst.
- This model mimics the oncogenic nature of the disease.
- The model can be used to study disease pathophysiology and test novel interventions.
