DMDMEASURED CORRECTION REFERENCE
Myogenic induction / DMD-locus correction
CTX-DMD-MYOGENIC-CORRECTION
- Source
- GSE272233
- Unit structure
- Biological repeat / sample; 3 repeats per group
Observed result21 samples; 24 background-specific dual-supported reversal pathways; 0 strict reversals shared across all 3 backgrounds.
Permitted use: DMD induction and locus-correction reference
Claim ceiling: No candidate-gene response prediction
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DMDMEASURED DISEASE REFERENCE
Patient skeletal muscle
CTX-DMD-PATIENT-MUSCLE
- Source
- PRJNA772047
- Unit structure
- Independent patient sample / pathway-level pseudobulk analysis
Observed result291 sample-level DMD disease pathways supported by CAMERA in the external patient-muscle reference.
Permitted use: External disease-pathway replication
Claim ceiling: No candidate-gene perturbation efficacy
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DMDMISSING REQUIRED OUTCOME
Candidate perturbation in DMD muscle
CTX-DMD-CANDIDATE-PERTURBATION
- Source
- No released outcome object
- Unit structure
- Required matched DMD/control biological repeats
Observed resultNo candidate has a qualifying independent perturbation outcome in a DMD muscle model yet.
Permitted use: Study design and predictability gating only
Claim ceiling: Prediction locked; absence is not a negative effect
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Patient myotube nuclei
CTX-FSHD-PATIENT-MYOTUBE
- Source
- GSE143452
- Unit structure
- 8 samples from 1 FSHD2 and 1 control donor
Observed result32,273 nuclei; all 17 requested DUX4-axis genes detected; donor-general prevalence is not estimable.
Permitted use: Descriptive state stratification
Claim ceiling: No donor-general or patient treatment estimate
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FSHDINDUCED CELL LINE RESPONSE
Inducible DUX4 myoblast time course
CTX-FSHD-IDUX4
- Source
- GSE205421
- Unit structure
- 4 culture wells per condition across 6 conditions
Observed result24 samples; 6,986 genes significant at 14 h using q < 0.05 and |log2FC| ≥ 1.
Permitted use: DUX4-response timing and experiment design
Claim ceiling: Cell-line response, not patient therapeutic efficacy
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DM1MEASURED CORRECTION REFERENCE
Isogenic repeat-corrected myoblast
CTX-DM1-ISOGENIC-MYOBLAST
- Source
- GSE127296
- Unit structure
- Independently derived clone; clone identity retained
Observed result8 clones; 163 differential splicing events at FDR < 0.05; clone effects remain a stated boundary.
Permitted use: Expression and splicing correction reference
Claim ceiling: Clones are not independent patients
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DM1MEASURED DISEASE REFERENCE
Patient skeletal muscle
CTX-DM1-PATIENT-MUSCLE
- Source
- GSE201255
- Unit structure
- Independent biopsy; repeat biopsies excluded
Observed result36 genes overlap the isogenic and patient splicing signals; global expression alignment is negligible (Spearman ρ = -0.039).
Permitted use: Patient expression/splicing comparison
Claim ceiling: No prognosis or treatment ranking
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SMAMEASURED INTERVENTION REFERENCE
Spinal organoid / ASO response
CTX-SMA-SPINAL-ORGANOID
- Source
- GSE290979 / GSE290980
- Unit structure
- Donor-derived line after technical aggregation
Observed result31 samples from 3 control, 2 SMA and 2 ASO-treated SMA lines; no global gene-expression reversal.
Permitted use: Human neural intervention-response design
Claim ceiling: No patient motor-outcome prediction
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SMAMEASURED RESIDUAL REFERENCE
Treated patient skeletal muscle
CTX-SMA-TREATED-MUSCLE
- Source
- GSE252128
- Unit structure
- Independent biopsy
Observed result8 treated SMA versus 7 control samples; 0 genes at q < 0.05 and |log2FC| ≥ 0.5; residual-module q-values exceed 0.30.
Permitted use: Residual muscle-module experiment design
Claim ceiling: Null threshold result is not proof of full correction
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SMAMEASURED DISEASE REFERENCE
Motor and cortical neurons
CTX-SMA-NEURONAL
- Source
- GSE302774
- Unit structure
- Independent culture replicate; n = 3 per condition and context
Observed resultKIF5A decreased concordantly in both neuronal contexts (log2FC approximately -1.31 in each).
Permitted use: Cross-neuronal disease-context comparison
Claim ceiling: No calibrated intervention or patient response
Open the source-bounded module →