Skip to main content
NMD-VCell Research OSDisease state → cell state → target → experiment
SearchLaunch Workspace
View: Plain
Release 2026.08DMD context observedDMD candidate-conditioned prediction not yet eligible
View scientific status
Evidence freeze: 3 August 2026Resource: v1.2.0-measured-dmd-evidenceSchema: 1.1Open release status
Explore NMD-VCell

Gene evidence summary

MON1A

Current recommendation: Test in human myogenic cells.

What we know

MON1A was experimentally perturbed in HepG2 cells.

What remains uncertain

Different DMD datasets do not fully agree on the disease-associated direction. A matched perturbation in a DMD-relevant muscle model is the truth needed to calibrate a future DMD-response model.

What should happen next

Test in human myogenic cells. Use independent reagents and biological replication, and predeclare one functional endpoint.

Current value: this record identifies the most informative next experiment and the result needed for model calibration. It does not yet establish a therapeutic or clinical conclusion.

Design this experimentReview research evidenceCompare evidenceOpen core data

Evidence journey

Where MON1A stands—and what creates the next evidence

Read left to right from released knowledge to translation. The highlighted next step is a research decision, not a target score.

Inspect datasets and methods →
01 · Database supportVersioned gene recordStable identity, provenance and downloadable evidence are available.
02 · Disease contextDMD context assembledSource-specific DMD signals are retained separately for interpretation.
03 · Model evidenceCalibration truth neededThe current evidence defines what a future DMD-response model must learn and test.
04 · Experimental perturbationObserved outside DMD muscleThe HepG2 response is useful for context transfer, not yet DMD truth.
05 · Functional phenotypeNext decision experimentTest in human myogenic cells
06 · Clinical evidenceFuture translation layerClinical interpretation follows reproducible disease-matched functional evidence.

Data behind this summary

Exact values currently available for MON1A

These values come from different biological contexts and are displayed separately. They are not combined into one target score.

Open all dataset rows →
HepG2 perturbation coverage94 cellsCells in the frozen aggregate; not biological replicates.
Skeletal-muscle expression8.05698 TPMGTEx v8 tissue-expression context; not perturbation evidence.
DMD source effects2/4 directions agree4 source-specific effects retained without pooling.
Human-myoblast fusion screenLFC -0.10707 · FDR 0.999999Assessed no-hit at FDR < 0.1; not evidence of no muscle function.

What this candidate is for

Does the MON1A response observed outside muscle transfer to a controlled human myogenic system?

A candidate record authorizes a bounded question and next experiment. It does not authorize a therapeutic or clinical claim.

Practical useTest context transfer and decide whether a matched DMD/control study is justified.
Decision available nowEligible for a preregistered human-myogenic feasibility experiment—not established as a therapeutic target.
What blocks itIndependent muscle-context perturbation is absent
Minimum next actionPerturb MON1A with at least two independent reagents and freeze one functional primary endpoint.
How would the result change the decision?

SupportiveAdvance to matched DMD/control validation only if engagement, QC, reagent concordance and biological replication all pass.

NullRecord a null muscle-context outcome and stop treating the non-muscle response as transferable when engagement and QC pass.

Inconclusive / QC failureResolve reagent, donor or batch discordance before any escalation.

Open Study CardPlan the experimentCompare decision gaps

Plain view presents the current story. Open Research for evidence details or Audit for machine fields and provenance.