NMD-VCell Neuromuscular Virtual Cell Research Platform Module: Data · Evidence → perturbation → experiment → outcome NMD = neuromuscular disorders

Release 2026.08DMD context observedDMD candidate-conditioned prediction not yet eligible

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Evidence freeze: 3 August 2026 Resource: v1.2.0-measured-dmd-evidence Schema: 1.1 Open release status →

Dataset Registry · governed subset

Which registered datasets can support which computational task?

This page contains 5 task-audited datasets selected from the broader 126-record Data Universe. It is a computational-role registry—not a claim that the platform contains only five datasets.

Product contract · DATASET-CARD-1.0

Dataset Card: What was measured, in which biological units, and what can it validly support?

Identity + versionDisease + cell contextDonor + statistical unitMatrix + metadata + hashesAccess + licenceReadiness + linked runs
Data estate12 workspace + 80 /share familiesStorage presence
Public inventory126 mapped recordsSearchable summaries
Task registry5 audited datasetsComputational roles assigned
DMD prediction trainingNot qualifiedMatched outcomes required
01SourceAccession, licence and provenance.
02Typed objectContext, unit, perturbation, time and endpoint.
03Training viewOnly compatible axes enter a model.
04HoldoutUnseen gene, donor, state or future batch.
No scalar readiness score. A dataset can qualify context, trajectory, disease correction or assay design without becoming direct evidence that a displayed candidate perturbation works in DMD.

Registry contract

Each selected dataset is audited against required metadata axes.

The Data Universe answers “what exists?”; this registry answers “what exact task may this object support?” Inclusion here is deliberately narrower than storage or analysis presence.

Open machine-readable registry →

Training outcome boundary: disease-context evidence, perturbation evidence and calibrated prediction truth remain separate fields.

Multi-axis readiness

Five datasets, five different computational roles.

Open full catalog →
DatasetDisease contextPerturbation outcomeCell-level outcomeTimeDonor / batchCurrent AI role
internal-release-substrateABSENT NOT DMDAVAILABLE SINGLE CRISPRIPARTIAL COMPONENT RESOLVEDSINGLE FROZEN ENDPOINTLIMITED RELEASED DIAGNOSTICSBASELINE READY SAME CONTEXT ONLY
GSE293514MUSCLE CONTEXT NOT DMD RESCUEAVAILABLE KNOCKOUT SCREEN ENDPOINTNOT RELEASED AS CELL POPULATION TRUTHPUBLICATION ENDPOINTNOT QUALIFIED FOR CURRENT MODELCONTEXT SCREEN ONLY
GSE272233AVAILABLE DMD CORRECTION REFERENCEDMD LOCUS CORRECTION NOT CANDIDATE PERTURBATIONABSENT BULK RNASINGLE STUDY ENDPOINTGROUPED SAMPLE IDENTITIES AVAILABLEORTHOGONAL REFERENCE ONLY
GSE52529ABSENT NON DISEASE REFERENCEABSENT UNPERTURBEDAVAILABLE SINGLE CELL REFERENCEAVAILABLE 0 24 48 72 HOURSNOT SUITABLE FOR UNSEEN DONOR PERTURBATIONTRAJECTORY REFERENCE ONLY
GSE133344ABSENT K562 METHOD REFERENCEAVAILABLE CRISPRA SINGLES AND PAIRSAVAILABLE PUBLIC METHOD REFERENCESINGLE STUDY ENDPOINTNOT A DONOR GENERALIZATION SETMETHOD CALIBRATION ONLY
internal-release-substrateObserved HepG2 CRISPRi candidate responsesBASELINE READY SAME CONTEXT ONLY
Disease / role
Context-limited; not DMD
Cell type
HepG2
Perturbation
CRISPRi single-gene
Time
Frozen endpoint aggregate
Size
21 governed candidate aggregates
Candidate coverage
21/21
Current AI roleTrain and audit the same-context baselineNo matched muscle or DMD perturbation outcome

Permitted use: Direct same-context perturbation observation

Internal release licence pending institutional approval

GSE293514Human myoblast fusion CRISPR screenCONTEXT SCREEN ONLY
Disease / role
Muscle context; not DMD rescue
Cell type
Human myoblast
Perturbation
Knockout screen
Time
Publication endpoint
Size
7,197 screen genes; 250 hits; 125 individually validated genes
Candidate coverage
9/21; 0 current fusion hits
Current AI roleQualify muscle-context coverage and endpoint compatibilityNot an independent candidate perturbation in a DMD model

Permitted use: Observed healthy-myoblast functional perturbation reference (Stage B0), not DMD replication

Source repository and publication terms apply; verify at accession

GSE272233DMD-locus CRISPR-correction transcript referenceORTHOGONAL REFERENCE ONLY
Disease / role
Duchenne muscular dystrophy
Cell type
Patient-derived and corrected cellular models
Perturbation
DMD-locus correction
Time
Study endpoint
Size
21 samples; 3 per reported group
Candidate coverage
Candidate transcripts observed; no candidate perturbation
Current AI roleCheck disease-correction direction and contextCandidate transcripts are observed but candidates were not perturbed

Permitted use: Orthogonal disease-correction reference

Source repository and publication terms apply; verify at accession

GSE52529Human myogenic differentiation time courseTRAJECTORY REFERENCE ONLY
Disease / role
Non-disease trajectory reference
Cell type
Primary human skeletal myoblast
Perturbation
None
Time
0, 24, 48 and 72 hours
Size
271 cells
Candidate coverage
20/21 measured
Current AI roleBuild an unperturbed human-myogenic state referenceNo perturbation-conditioned trajectory and no longitudinal DMD state

Permitted use: Unperturbed trajectory reference only

Source repository and package terms apply; verify at accession

GSE133344CRISPRa single and combinatorial calibration referenceMETHOD CALIBRATION ONLY
Disease / role
Non-muscle method calibration
Cell type
K562
Perturbation
CRISPRa singles and pairs
Time
Study endpoint
Size
287 perturbations; 112 singles; 132 pairs
Candidate coverage
20/21 present as expression features
Current AI roleCalibrate activation and combination task designNo matched muscle or DMD activation response for current candidates

Permitted use: Method calibration only; not muscle/DMD overexpression evidence

Source repository and publication terms apply; verify at accession