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Updated: Sep 25, 2025

Investigating Target Gene Function in a CD40 Agonistic Antibody-induced Colitis Model using CRISPR/Cas9-based Technologies
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Linking Genetic Diagnosis to Therapeutic Approach in Very Early Onset Inflammatory Bowel Disease: Pharmacologic

Anne E Levine1,2, Hengqi B Zheng1,2, David L Suskind3,4

  • 1Division of Gastroenterology, Seattle Children's Hospital Inflammatory Bowel Disease Center, Seattle, WA, USA.

Paediatric Drugs
|April 25, 2022
PubMed

Insights

Very early onset inflammatory bowel disease (VEO-IBD) can stem from genetic causes. This review covers monogenic VEO-IBD classifications and precision medicine therapies, including immune modulation and transplantation.

Area of Science:

  • Pediatric Gastroenterology
  • Immunology
  • Genetics

Background:

  • Very early onset inflammatory bowel disease (VEO-IBD) affects children under six.
  • A subset of VEO-IBD cases result from identifiable monogenic causes.
  • Advances in genetic testing have identified approximately 100 genes linked to monogenic VEO-IBD.

Purpose of the Study:

  • To review classifications of monogenic disorders causing VEO-IBD.
  • To discuss current and emerging targeted therapies for VEO-IBD.
  • To provide an overview of precision medicine approaches for VEO-IBD.

Main Methods:

  • Literature review of genetic testing and VEO-IBD.
  • Analysis of immune pathways implicated in monogenic VEO-IBD.
  • Compilation of current therapeutic strategies for VEO-IBD.

Main Results:

  • Monogenic VEO-IBD involves diverse immune pathways.
  • Genetic testing enables more accurate VEO-IBD diagnosis.
  • Targeted therapies offer precision medicine avenues for VEO-IBD.

Conclusions:

  • Monogenic VEO-IBD presents diverse genetic underpinnings.
  • Targeted pharmacologic treatments are emerging for VEO-IBD.
  • A multi-modal approach including nutrition, surgery, and transplantation is key.

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