NMD-VCell Evidence & Experiment Workbench Audit DMD evidence, compare gaps, and freeze the missing test Module: Registry · evidence-to-experiment workflow NMD = neuromuscular disorders
Search Data & API Current release

Current limit: the site can compare existing evidence, but no candidate has yet been independently repeated in a DMD muscle model.

Research evidence only 21 prioritized candidate perturbation records 2,160 benchmark perturbation targets 250 observed human-myoblast fusion hits Prospective DMD candidate validation pending Boundary & release
21 Workbench candidates are surfaced for consideration, not ranked as validated targets 2,160 target-level perturbation responses form the frozen HepG2 benchmark substrate GSE293514 Stage B0 contributes 250 healthy-human-myoblast fusion hits and 125 individually validated genes No validated DMD perturbation prediction v1.2.0-measured-dmd-evidence Frozen 3 August 2026 Schema 1.1 Model ridge-safe-v2.3 Benchmark repeated-fold-v2.2 Build EA-20260804-49 DOI pending Open evidence boundary →

Measured DMD evidence · Stage C2

DMD induction, patient replication and CRISPR correction are now connected by an auditable pathway analysis.

This release adds biological-repeat-aware Reactome tests for GSE272233 and sample-level external disease validation. It strengthens disease-context evidence and provides reference axes for a prespecified prospective candidate-validation study.

Open API summary Download Figure 8 Verify objects
Measured disease context, prediction still lockedDMD-locus perturbation and patient-muscle pathway evidence are observed. Candidate-gene response prediction, therapeutic efficacy and patient-level simulation remain unavailable.

Evidence chain

Four layers are shown together, but their inferential roles remain separate.

Inspect statistical units →
DMD induction

Engineered and patient DMD agree descriptively

GSE233606 provides an unselected gene-axis bridge between engineered DMD and a patient line. It is descriptive, not donor-level inference.

Patient replication

291 camera-supported pathways

PRJNA772047 uses five reported donor/sample pseudobulks. PRJNA1218493 and mouse GSE156497 remain directional sensitivity layers.

CRISPR correction

24 background-specific reversals

GSE272233 uses three reported biological repeats per group. No dual-supported pathway reverses across all three mutation backgrounds.

Organoid heterogeneity

Context dependence retained

Organoid evidence is shown as a heterogeneity layer and is not pooled into a universal DMD response.

NAR main figure

Disease induction → patient replication → correction → heterogeneity

Download source data →
Four-panel measured DMD evidence figure showing induction, external patient pathway agreement, CRISPR correction and organoid heterogeneity.
Figure 8 keeps descriptive, formal and sensitivity-only evidence visually distinct. The lower strip summarizes the four measured evidence layers and their inferential units.

Primary statistical readout

Strict consensus is sparse, and that result is preserved.

Dual reversal in all three correction backgrounds0
Strict independent reversal in all three backgrounds0
Background-specific dual-supported reversals24
Patient pathways with camera support291

The 492 fixed-effect cross-background reversals are explicitly exploratory because WT is non-isogenic and heterogeneity is substantial. They are not promoted to validated virtual-cell predictions.

Reusable objects

API, pathway tables, sample QC and code share one release identity.

GSE272233 API External validation API Sample QC API camera results External effects

Independent rebuild receipt

All 16 clean-room comparisons pass.

The path-isolated rebuild regenerated GSE233606 induction, GSE272233 correction and camera tests, three external pseudobulk pathway layers, GSE277637 heterogeneity and Figure 8. Numeric tables and metadata-independent figure pixels match the released analysis.

Open clean-room API Download report Download audit

Boundary: this is path/process-isolated computational reproduction using the same system Python/R packages, not a container-level independent-host reproduction and not biological validation.

Claim boundary

Supported: Measured DMD-locus induction/correction and sample-level external disease pathway replication with explicit small-n limitations.

Not supported: Candidate-gene response prediction in DMD cells, therapeutic efficacy, patient-level simulation and clinical decision support.