Journal of Crohn's & colitis
Cure8 research brief
Why This Matters
This review collects evidence about treatments specifically studied in monogenic forms of IBD, which can point toward precision, gene- or pathway-targeted therapies for people whose disease is caused by a single genetic defect. That matters because standard IBD trial data often do not apply to these rare conditions.
Who Should Pay Attention
Clinicians and researchers working on genetic or pediatric IBD; patients and caregivers of children or adults with confirmed monogenic IBD; specialists considering targeted or curative approaches such as stem-cell transplant or gene therapy.
Study Snapshot
Story typeResearch paper
Evidence typeSystematic review
Source depthJournal abstract
What To Know
This paper is a systematic review that compiles published evidence about treatments used for monogenic inflammatory bowel disease (mIBD). The authors evaluated reports covering 35 different therapeutics across 102 genetic causes of mIBD, summarizing gene–drug response pairs from case series and case reports.
The review found at least one effective pharmacologic intervention for about 61% of the named monogenic conditions, but many conditions still lack an established effective therapy.
Treatments with gene- or pathway-specific responses included allogeneic hematopoietic stem cell transplantation, gene therapy, anti‑TNF agents, IL‑1 inhibitors, mTOR inhibitors, eculizumab for CD55 deficiency, abatacept for CTLA4 deficiency, and an immunometabolic agent (empagliflozin) in glycogen storage disease type 1b.
Because many data come from individual case reports, case series, and small cohorts, the review emphasizes precision-medicine approaches tailored to the genetic or pathway mechanism rather than standard randomized-trial evidence.
If you or someone you care for has a confirmed monogenic cause of IBD, this review suggests discussing mechanism-targeted options with a specialist and considering referral to centers with experience in rare genetic IBD.
Keep In Mind
Structured-content depth is an abstract from a systematic review published in Journal of Crohn’s & Colitis. Much of the evidence summarized is from case reports, case series, and small observational studies; few randomized trials exist for these rare conditions.
The review highlights both promising gene- or pathway-specific responses and significant gaps where no effective therapy is yet identified.
Source Details
Review the original publication for the complete reporting, methods, and context.
Systematic review
Evidence type derived from source or registry metadata.
PublicationJournal of Crohn's & colitis
AuthorsYeh PJ, Charlesworth JEG, Taylor H +19 more
Study typeIm, systematic review, journal article
Indexed viaEurope PMC
Source typeResearch paper
PublishedSep 1, 2026, 12:00 AM
Content availableJournal abstract
Funding disclosed by the source: Helmsley Charitable Trust; National Institute for Health and Care Research (NIHR) Oxford Biomedical Research Centre (BRC)
This Cure8 brief is based on source text from the linked article. Cure8 is informational only and is not a substitute for professional medical advice, diagnosis, or treatment.
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