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 →

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Dataset Card · nmd-vcell-dataset-registry-record/1.0

CRISPRa single and combinatorial calibration reference

A versioned, citable account of what was measured, which task this object may support and which prediction claims remain blocked.

01SourceGSE133344
02Typed contextK562 method-calibration reference
03Qualified roleCalibrate activation and combination task design
04DMD prediction outcomeNot qualified

What it proves

CRISPRa single and pair perturbation design can be calibrated in a public single-cell method reference.

single cell · Single-cell gene expression

What it cannot prove

It cannot support muscle/DMD overexpression claims or infer combination benefit for current candidates.

Dataset value is task-specific; no Dataset Card alone is evidence of candidate efficacy, patient response or clinical utility.

Best model use

Use for activation-design and pair-task calibration outside the DMD biological claim space.

Calibrate activation and combination task design

Next experiment handoff

Move activation or pair hypotheses into a muscle/DMD-specific assay before interpreting biological effect.

No matched muscle or DMD activation response for current candidates

Identity & access

Source receipt

Accession
GSE133344
Licence
Source repository and publication terms apply; verify at accession
Source verification
required
Stable page
/resource/dataset/gse133344/

Biological context

What it represents

Disease / context
Non-muscle method calibration
Species
Homo sapiens
Tissue / model
K562 method-calibration reference
Cell type
K562
Donor metadata
not a donor generalization set

Measurement design

What was measured

Modality
single-cell perturbation expression
Observation unit
single cell
Size
287 perturbations; 112 singles; 132 pairs
Count state
public method reference
Endpoint
Single-cell gene expression

Perturbation design

Intervention contract

Perturbation
CRISPRa singles and pairs
Dose
CRISPRa singles and pairs; dose not harmonized in current object
Timepoints
Study endpoint
Direct DMD candidate outcome
No

Replication & provenance

Receipt completeness

Biological replicates
source publication required
Technical replicates
source publication required
Batch / platform
source publication required
Raw hashes
upstream accession required
Processed hashes
processed feature table checksummed
Analysis hashes
derived calibration objects available

Benchmark partition

Permitted computational use

Method calibration only; not muscle/DMD overexpression evidence

  • G7_method_calibration_reference_only
Leakage grouping
method calibration only not dmd training
Donor holdout
No
Disease-state holdout
No
Prospective holdout
No

Readiness decision

Calibrate activation and combination task design

No matched muscle or DMD activation response for current candidates

Associated resources

Continue through the governed loop.