What it proves
DMD-locus correction changes disease-context transcript programs in patient-derived/corrected models.
bulk sample · Bulk RNA expression and correction contrasts
Dataset Card · nmd-vcell-dataset-registry-record/1.0
DMD-locus CRISPR-correction transcript reference
A versioned, citable account of what was measured, which task this object may support and which prediction claims remain blocked.
01SourceGSE272233
→02Typed contextDMD-locus correction reference
→03Qualified roleCheck disease-correction direction and context
→04DMD prediction outcomeNot qualified
What it cannot prove
It cannot isolate a displayed candidate's perturbation effect because those candidates were not perturbed.
Dataset value is task-specific; no Dataset Card alone is evidence of candidate efficacy, patient response or clinical utility.
Best model use
Use as orthogonal DMD directionality and disease-correction context, not as candidate-training truth.
Check disease-correction direction and context
Next experiment handoff
Use the corrected-vs-DMD contrast to choose endpoints for a separate candidate perturbation study.
Candidate transcripts are observed but candidates were not perturbed
Identity & access
Source receipt
- Accession
- GSE272233
- Licence
- Source repository and publication terms apply; verify at accession
- Source verification
- required
- Stable page
/resource/dataset/gse272233/
Biological context
What it represents
- Disease / context
- Duchenne muscular dystrophy
- Species
- Homo sapiens
- Tissue / model
- DMD-locus correction reference
- Cell type
- Patient-derived and corrected cellular models
- Donor metadata
- grouped sample identities available
Measurement design
What was measured
- Modality
- bulk RNA expression
- Observation unit
- bulk sample
- Size
- 21 samples; 3 per reported group
- Count state
- not single cell
- Endpoint
- Bulk RNA expression and correction contrasts
Perturbation design
Intervention contract
- Perturbation
- DMD-locus correction
- Dose
- DMD-locus correction; candidate perturbation absent
- Timepoints
- Study endpoint
- Direct DMD candidate outcome
- No
Replication & provenance
Receipt completeness
- Biological replicates
- 3 per reported group
- Technical replicates
- source publication required
- Batch / platform
- source publication required
- Raw hashes
- upstream accession required
- Processed hashes
- processed matrix checksummed
- Analysis hashes
- derived tables in resource manifest
Benchmark partition
Permitted computational use
Orthogonal disease-correction reference
disease_correction_context_only
- Leakage grouping
- orthogonal reference no candidate training
- Donor holdout
- No
- Disease-state holdout
- No
- Prospective holdout
- No
Readiness decision
Check disease-correction direction and context
Candidate transcripts are observed but candidates were not perturbed
Associated resources