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Official PDF TranslationStem Cell Research & Therapy

Toward clinically relevant models of complex perianal fistulas: refining preclinical evaluation for exosome-based therapies

Authors: Qianqian Ye; Qingming Wang

DOI: 10.1186/s13287-025-04429-9Status: Verified Translated Edition
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Key Findings in This Report

• The study validates hUCMSC-derived exosomes as a promising minimally invasive therapy for complex perianal fistulas, acting via HIF-1α/TGF-β/Smad pathway and microbiota modulation. • Rodent models have anatomical and microbiota disparities that limit clinical translation; large-animal models (porcine, canine) are recommended for validation. • Long-term safety of exosome therapy, including potential off-target effects and immune priming, requires systematic evaluation before clinical adoption. • Refined preclinical models should incorporate extended fistula retention, repeated microbial stimulation, and advanced imaging (contrast-enhanced MRI) to better mimic chronic human disease.