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

Current limit: disease modules compare measured states and intervention evidence, but none emits a calibrated patient-level prediction. DMD candidate validation remains prospective.

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 →

Disease evidence module · multidisease-evidence-v0.2.0

DMD · Duchenne muscular dystrophy

Measured DMD induction and locus correction are connected to a governed 21-candidate prospective test queue.

deep_measured_context_with_explicit_candidate_truth_gap
Open module APIOpen full DMD evidence workbench
Unified v0.2 contractstrict state + qualifiers · stable candidate IDs · statistical unit · provenance · next experimentJSON Schema
Correction dataset21 samples · 3 repeats/group
External patient muscle3 DMD + 2 normal · small-n
Candidate queue21 governed actions · no numeric current rank
Measured candidate truth0/21 · prospective

Interpretation and boundary

Positive, null and missing evidence remain separate.

DMD induction/correction and independent patient-muscle pathways are measured, but direct candidate perturbation response is 0/21. The current candidate list is therefore a governed next-action queue, not a validated target ranking.

Selected evidence figure

Measured context and claim ceiling stay visible together.

DMD measured evidence and explicit claim boundary.

Dataset registry

Each object declares its biological/statistical unit.

MEASUREDGSE233606 ↗
DESCRIPTIVE_OR_DIRECTIONAL

human single-cell RNA-seq

healthy; patient DMD; engineered DMD; 3566 cells

Unit: one line per condition
Role: measured DMD induction and patient-line replication
MEASUREDPRJNA772047 ↗

human single-nucleus RNA-seq pseudobulk

3 DMD; 2 Normal; 8776 nuclei; 5 pseudobulks

Unit: 5 reported donors/samples
Role: independent patient-muscle pathway replication
MEASUREDPRJNA1218493 ↗
DESCRIPTIVE_OR_DIRECTIONAL

human single-cell/nucleus RNA-seq pseudobulk

2 DMD; 1 Normal; 7439 cells; 3 pseudobulks

Unit: 3 reported samples
Role: external disease-direction sensitivity
MEASUREDGSE156497 ↗
DESCRIPTIVE_OR_DIRECTIONAL

mouse single-cell RNA-seq pseudobulk

1 DMD-like; 1 WT; 11581 cells; 2 pseudobulks

Unit: 2 reported samples
Role: cross-species disease-direction sensitivity
CORRECTEDGSE272233 ↗
DMD_LOCUS_CORRECTION

human bulk RNA-seq

WT; 3 DMD duplication backgrounds; corrected clones; 21 samples

Unit: 3 reported biological repeats per group
Role: measured DMD-locus CRISPR correction
MEASUREDGSE277637 ↗
DESCRIPTIVE_OR_DIRECTIONAL

human organoid single-cell RNA-seq pseudobulk

3 DMD patient lines; 1 WT line; 10480 cells; 4 pseudobulks

Unit: 4 lines with one shared WT
Role: model-system heterogeneity stress test
MISSINGPROSPECTIVE-CANDIDATE-PERTURBSEQ ↗
PRESPECIFIEDZERO_OF_21_CANDIDATE_RESPONSES_OBSERVED

DMD-relevant muscle perturbation experiment

21 candidates; matched controls and functional endpoints prespecified; outcomes not yet observed

Unit: future independent biological replicate / donor or line
Role: candidate-level DMD response truth

Candidate establishment

Role-typed objects link directly to the next missing experiment.

ObjectRoleState + qualifiersPermitted interpretation / next experiment
CPEB1
NMDVCELL:DMD:CANDIDATE:CPEB1
Identifiers & provenanceHGNC:21744 · ENSG00000214575
Datasets: none — prospective gap
Statistical unit: not yet observed for candidate-level DMD response
prospective_candidatePREDICTED
HYPOTHESIS_TRIAGENEXT_ACTION_QUEUENO_MEASURED_DMD_CANDIDATE_RESPONSE
Governed prospective candidate and next action; not a validated target or treatment rank.
Next missing experimentVerify expression in the intended DMD muscle model before perturbation.
DNAAF3
NMDVCELL:DMD:CANDIDATE:DNAAF3
Identifiers & provenanceHGNC:30492 · ENSG00000167646
Datasets: none — prospective gap
Statistical unit: not yet observed for candidate-level DMD response
prospective_candidatePREDICTED
HYPOTHESIS_TRIAGENEXT_ACTION_QUEUENO_MEASURED_DMD_CANDIDATE_RESPONSE
Governed prospective candidate and next action; not a validated target or treatment rank.
Next missing experimentVerify expression in the intended DMD muscle model before perturbation.
EPS8L1
NMDVCELL:DMD:CANDIDATE:EPS8L1
Identifiers & provenanceHGNC:21295 · ENSG00000131037
Datasets: none — prospective gap
Statistical unit: not yet observed for candidate-level DMD response
prospective_candidatePREDICTED
HYPOTHESIS_TRIAGENEXT_ACTION_QUEUENO_MEASURED_DMD_CANDIDATE_RESPONSE
Governed prospective candidate and next action; not a validated target or treatment rank.
Next missing experimentVerify expression in the intended DMD muscle model before perturbation.
ZFP69B
NMDVCELL:DMD:CANDIDATE:ZFP69B
Identifiers & provenanceHGNC:28053 · ENSG00000187801
Datasets: none — prospective gap
Statistical unit: not yet observed for candidate-level DMD response
prospective_candidatePREDICTED
HYPOTHESIS_TRIAGENEXT_ACTION_QUEUENO_MEASURED_DMD_CANDIDATE_RESPONSE
Governed prospective candidate and next action; not a validated target or treatment rank.
Next missing experimentVerify expression in the intended DMD muscle model before perturbation.
INTS13
NMDVCELL:DMD:CANDIDATE:INTS13
Identifiers & provenanceHGNC:20174 · ENSG00000064102
Datasets: none — prospective gap
Statistical unit: not yet observed for candidate-level DMD response
prospective_candidatePREDICTED
HYPOTHESIS_TRIAGENEXT_ACTION_QUEUENO_MEASURED_DMD_CANDIDATE_RESPONSE
Governed prospective candidate and next action; not a validated target or treatment rank.
Next missing experimentResolve dependency, toxicity and context-risk flags before DMD perturbation.
NAGLU
NMDVCELL:DMD:CANDIDATE:NAGLU
Identifiers & provenanceHGNC:7632 · ENSG00000108784
Datasets: none — prospective gap
Statistical unit: not yet observed for candidate-level DMD response
prospective_candidatePREDICTED
HYPOTHESIS_TRIAGENEXT_ACTION_QUEUENO_MEASURED_DMD_CANDIDATE_RESPONSE
Governed prospective candidate and next action; not a validated target or treatment rank.
Next missing experimentResolve dependency, toxicity and context-risk flags before DMD perturbation.
RAC3
NMDVCELL:DMD:CANDIDATE:RAC3
Identifiers & provenanceHGNC:9803 · ENSG00000169750
Datasets: none — prospective gap
Statistical unit: not yet observed for candidate-level DMD response
prospective_candidatePREDICTED
HYPOTHESIS_TRIAGENEXT_ACTION_QUEUENO_MEASURED_DMD_CANDIDATE_RESPONSE
Governed prospective candidate and next action; not a validated target or treatment rank.
Next missing experimentResolve dependency, toxicity and context-risk flags before DMD perturbation.
RNASEH2C
NMDVCELL:DMD:CANDIDATE:RNASEH2C
Identifiers & provenanceHGNC:24116 · ENSG00000172922
Datasets: none — prospective gap
Statistical unit: not yet observed for candidate-level DMD response
prospective_candidatePREDICTED
HYPOTHESIS_TRIAGENEXT_ACTION_QUEUENO_MEASURED_DMD_CANDIDATE_RESPONSE
Governed prospective candidate and next action; not a validated target or treatment rank.
Next missing experimentResolve dependency, toxicity and context-risk flags before DMD perturbation.
RNF8
NMDVCELL:DMD:CANDIDATE:RNF8
Identifiers & provenanceHGNC:10071 · ENSG00000112130
Datasets: none — prospective gap
Statistical unit: not yet observed for candidate-level DMD response
prospective_candidatePREDICTED
HYPOTHESIS_TRIAGENEXT_ACTION_QUEUENO_MEASURED_DMD_CANDIDATE_RESPONSE
Governed prospective candidate and next action; not a validated target or treatment rank.
Next missing experimentResolve dependency, toxicity and context-risk flags before DMD perturbation.
WDR4
NMDVCELL:DMD:CANDIDATE:WDR4
Identifiers & provenanceHGNC:12756 · ENSG00000160193
Datasets: none — prospective gap
Statistical unit: not yet observed for candidate-level DMD response
prospective_candidatePREDICTED
HYPOTHESIS_TRIAGENEXT_ACTION_QUEUENO_MEASURED_DMD_CANDIDATE_RESPONSE
Governed prospective candidate and next action; not a validated target or treatment rank.
Next missing experimentResolve dependency, toxicity and context-risk flags before DMD perturbation.
ZNF236
NMDVCELL:DMD:CANDIDATE:ZNF236
Identifiers & provenanceHGNC:13028 · ENSG00000130856
Datasets: none — prospective gap
Statistical unit: not yet observed for candidate-level DMD response
prospective_candidatePREDICTED
HYPOTHESIS_TRIAGENEXT_ACTION_QUEUENO_MEASURED_DMD_CANDIDATE_RESPONSE
Governed prospective candidate and next action; not a validated target or treatment rank.
Next missing experimentResolve dependency, toxicity and context-risk flags before DMD perturbation.
MON1A
NMDVCELL:DMD:CANDIDATE:MON1A
Identifiers & provenanceHGNC:28207 · ENSG00000164077
Datasets: none — prospective gap
Statistical unit: not yet observed for candidate-level DMD response
prospective_candidatePREDICTED
HYPOTHESIS_TRIAGENEXT_ACTION_QUEUENO_MEASURED_DMD_CANDIDATE_RESPONSE
Governed prospective candidate and next action; not a validated target or treatment rank.
Next missing experimentRun a replicate-aware DMD muscle perturbation assay with prespecified molecular and functional endpoints.
ZNF133
NMDVCELL:DMD:CANDIDATE:ZNF133
Identifiers & provenanceHGNC:12917 · ENSG00000125846
Datasets: none — prospective gap
Statistical unit: not yet observed for candidate-level DMD response
prospective_candidatePREDICTED
HYPOTHESIS_TRIAGENEXT_ACTION_QUEUENO_MEASURED_DMD_CANDIDATE_RESPONSE
Governed prospective candidate and next action; not a validated target or treatment rank.
Next missing experimentRun a replicate-aware DMD muscle perturbation assay with prespecified molecular and functional endpoints.
ADAM10
NMDVCELL:DMD:CANDIDATE:ADAM10
Identifiers & provenanceHGNC:188 · ENSG00000137845
Datasets: none — prospective gap
Statistical unit: not yet observed for candidate-level DMD response
prospective_candidatePREDICTED
HYPOTHESIS_TRIAGENEXT_ACTION_QUEUENO_MEASURED_DMD_CANDIDATE_RESPONSE
Governed prospective candidate and next action; not a validated target or treatment rank.
Next missing experimentReplicate the source-context signal in a frozen independent computational audit before wet-lab escalation.
CALR
NMDVCELL:DMD:CANDIDATE:CALR
Identifiers & provenanceHGNC:1455 · ENSG00000179218
Datasets: none — prospective gap
Statistical unit: not yet observed for candidate-level DMD response
prospective_candidatePREDICTED
HYPOTHESIS_TRIAGENEXT_ACTION_QUEUENO_MEASURED_DMD_CANDIDATE_RESPONSE
Governed prospective candidate and next action; not a validated target or treatment rank.
Next missing experimentReplicate the source-context signal in a frozen independent computational audit before wet-lab escalation.
DDX19B
NMDVCELL:DMD:CANDIDATE:DDX19B
Identifiers & provenanceHGNC:2742 · ENSG00000157349
Datasets: none — prospective gap
Statistical unit: not yet observed for candidate-level DMD response
prospective_candidatePREDICTED
HYPOTHESIS_TRIAGENEXT_ACTION_QUEUENO_MEASURED_DMD_CANDIDATE_RESPONSE
Governed prospective candidate and next action; not a validated target or treatment rank.
Next missing experimentReplicate the source-context signal in a frozen independent computational audit before wet-lab escalation.
DNM1
NMDVCELL:DMD:CANDIDATE:DNM1
Identifiers & provenanceHGNC:2972 · ENSG00000106976
Datasets: none — prospective gap
Statistical unit: not yet observed for candidate-level DMD response
prospective_candidatePREDICTED
HYPOTHESIS_TRIAGENEXT_ACTION_QUEUENO_MEASURED_DMD_CANDIDATE_RESPONSE
Governed prospective candidate and next action; not a validated target or treatment rank.
Next missing experimentReplicate the source-context signal in a frozen independent computational audit before wet-lab escalation.
EHMT2
NMDVCELL:DMD:CANDIDATE:EHMT2
Identifiers & provenanceHGNC:14129 · ENSG00000204371
Datasets: none — prospective gap
Statistical unit: not yet observed for candidate-level DMD response
prospective_candidatePREDICTED
HYPOTHESIS_TRIAGENEXT_ACTION_QUEUENO_MEASURED_DMD_CANDIDATE_RESPONSE
Governed prospective candidate and next action; not a validated target or treatment rank.
Next missing experimentReplicate the source-context signal in a frozen independent computational audit before wet-lab escalation.
LMO2
NMDVCELL:DMD:CANDIDATE:LMO2
Identifiers & provenanceHGNC:6642 · ENSG00000135363
Datasets: none — prospective gap
Statistical unit: not yet observed for candidate-level DMD response
prospective_candidatePREDICTED
HYPOTHESIS_TRIAGENEXT_ACTION_QUEUENO_MEASURED_DMD_CANDIDATE_RESPONSE
Governed prospective candidate and next action; not a validated target or treatment rank.
Next missing experimentReplicate the source-context signal in a frozen independent computational audit before wet-lab escalation.
MPHOSPH6
NMDVCELL:DMD:CANDIDATE:MPHOSPH6
Identifiers & provenanceHGNC:7214 · ENSG00000135698
Datasets: none — prospective gap
Statistical unit: not yet observed for candidate-level DMD response
prospective_candidatePREDICTED
HYPOTHESIS_TRIAGENEXT_ACTION_QUEUENO_MEASURED_DMD_CANDIDATE_RESPONSE
Governed prospective candidate and next action; not a validated target or treatment rank.
Next missing experimentReplicate the source-context signal in a frozen independent computational audit before wet-lab escalation.
GFOD2
NMDVCELL:DMD:CANDIDATE:GFOD2
Identifiers & provenanceHGNC:28159 · ENSG00000141098
Datasets: none — prospective gap
Statistical unit: not yet observed for candidate-level DMD response
prospective_candidatePREDICTED
HYPOTHESIS_TRIAGENEXT_ACTION_QUEUENO_MEASURED_DMD_CANDIDATE_RESPONSE
Governed prospective candidate and next action; not a validated target or treatment rank.
Next missing experimentHold the candidate until the named external evidence audit is complete.
Supports
  • measured DMD induction and locus correction
  • small-n patient-muscle pathway replication
  • prospective candidate experiment design
Does not support
  • measured candidate-level DMD response
  • therapeutic efficacy
  • patient-level simulation or clinical recommendation

Machine-readable module

Summary, datasets, candidate logic and claim boundary are separate objects.

summary · datasets · candidates · boundary