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Gene Therapy in Crohn's Disease: Current Preclinical Challenges and Future Translational Avenues

Solafah Abdalla1,2, Antoine Brouquet1,2, Romina Aron-Badin3

  • 1Service de Chirurgie Viscérale et Oncologique, Hôpital de Bicêtre, Assistance Publique-Hôpitaux de Paris, 94270 Paris, France.

Biomedicines
|December 30, 2025
PubMed

Insights

Preclinical gene therapy for Crohn

Area of Science:

  • Gastroenterology
  • Molecular Therapy
  • Immunology

Background:

  • Crohn's disease (CD) is a complex inflammatory bowel disease (IBD) with limited effective treatments.
  • Preclinical gene therapy for CD faces challenges in target identification, delivery to resistant cells, and suitable animal models.

Purpose of the Study:

  • To review and critically assess preclinical gene therapy studies for Crohn's disease and related IBDs.
  • To evaluate vector platforms, biodistribution, and therapeutic strategies, with a focus on the enteric nervous system (ENS).

Main Methods:

  • Systematic review of preclinical gene therapy studies for CD and IBD.
  • Analysis of vector types, promoters, and administration routes in rodent and non-human primate models.
  • Assessment of strategies targeting the ENS and therapeutic aims.

Main Results:

  • Gene therapy approaches show potential for attenuating intestinal inflammation and preventing CD recurrence.
  • Biodistribution and efficacy data vary significantly based on vector platform and delivery method.
  • Targeting the ENS presents unique challenges and opportunities for CD treatment.

Conclusions:

  • A foundational framework is established to guide translational research in gene therapy for CD and IBD.
  • Further development of robust models and targeted delivery systems is crucial for clinical translation.
  • Gene therapy offers promising avenues for managing intestinal inflammation and postoperative recurrence in CD.
Keywords:
Crohn’s diseaseenteric nervous systemgene therapy

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