v1.0.0-database-resourceSchema 1.1Model ridge-safe-v2.3Benchmark repeated-fold-v2.2Build EA-20260729-15v1.0 is a database and evidence-governance release; it is not a validated disease-prediction or clinical decision-support release.
REGRegistered Study Card5 open fields before registration
b
Bioinformatics result snapshot
Legacy integrated DMD priorup
Source agreement50%
Skeletal-muscle TPM8.06
External screenassessed no hit
Dependency cautionnot flagged
DepMap effect-0.111
c
Evidence-to-experiment route
Context
Human myoblast or myotube model; healthy/DMD/isogenic background and differentiation state declared before registration.
Perturb
CRISPRi, CRISPRa or siRNA selected for the biological question; at least two independent reagents where feasible.
Endpoint
One predeclared functional muscle endpoint such as fusion, morphology or contractile readout.
Infer
Independent biological replicate or independently generated perturbation unit; cells within one aggregate are not inferential replicates.
Model
Mixed-effects model with treatment fixed effect and donor/batch/reagent blocking; report reagent-specific estimates.
d
Registration gap map
direction rationale
numeric minimally important effect
numeric negligible-effect margin
QC thresholds
final powered sample size
These bars are field-state indicators only. They do not create a target score, rank, cluster or prediction claim.
Predeclared design scaffold
Biological context
Human myoblast or myotube model; healthy/DMD/isogenic background and differentiation state declared before registration.
Perturbation modality
CRISPRi, CRISPRa or siRNA selected for the biological question; at least two independent reagents where feasible.
Primary endpoint
One predeclared functional muscle endpoint such as fusion, morphology or contractile readout.
Primary estimand
Between-condition mean difference (or prespecified ratio) in the primary functional endpoint at the frozen time point.
Inferential unit
Independent biological replicate or independently generated perturbation unit; cells within one aggregate are not inferential replicates.
Planning sample size
3–6 independent biological replicates per condition and reagent as a pilot planning range; final n requires assay variance and minimally important effect.
Randomisation unit
Independent culture well or biological replicate, not individual cells.
Statistical model
Mixed-effects model with treatment fixed effect and donor/batch/reagent blocking; report reagent-specific estimates.
Decision-blocking gap
GAP-02 Independent muscle-context perturbation is absent
Current selection: unresolved requires registration
State whether the disease-associated direction is hypothesized as causal, compensatory or accompanying, and preserve the opposite-direction alternative. DMD direction and counteralignment never choose an intervention automatically.
Allowed registered hypotheses: activation; inhibition; bidirectional exploration; direction not identifiable.
Comparator and controls
Non-targeting control.
Positive assay control.
Mock delivery control when delivery itself can affect phenotype.
Target-engagement and viability controls.
Secondary endpoints
Target engagement.
Viability/toxicity.
Directional transcriptomic response.
State-transition extension
Mechanism hypothesis and registered time axis
Scientific object: perturbation × cell state × disease context × time × phenotype
Required before registration: state the proposed early molecular mediator, the expected cell-state transition and the downstream functional consequence.
6–12 hearly molecular or signalling response
planning default requires assay calibration
24–48 hregulatory program and cell-state transition
planning default requires assay calibration
4–7 ddifferentiation and functional phenotype
planning default requires assay calibration
Cell context fields
Required before registration: healthy, DMD or isogenic corrected. Required before registration: proliferating myoblast, early differentiation, fusion or maturing myotube. Required before registration; preserve donor-specific estimates.
Cell–cell consequence
Current status: not assessed. Future levels: conditioned medium; two-cell co-culture; three-dimensional muscle model; spatial perturbation model.
Blocking factors
Donor/isogenic pair.
Differentiation batch.
Plate and reagent identity.
Assay QC thresholds
Target-engagement threshold must be numeric and frozen.
Viability floor and image-quality thresholds must be numeric and frozen.
Minimally important effect
Required before registration; derive from assay biology or a justified pilot and store the numeric value with units.
Negligible-effect margin
Required before interpreting a null result; store a symmetric or asymmetric numeric margin with units.
Multiplicity and missing data
Primary endpoint across the two reagents; secondary endpoints form a separate multiplicity family.
Define exclusions before unblinding; report all missing units and reasons; do not single-impute primary outcomes without a prespecified sensitivity analysis.
Replication rules
Both reagents must agree in direction and neither may fail the engagement/QC gate; discordance is inconclusive.
Direction must replicate beyond a single donor or isogenic pair before L4 escalation.
Stop rules
Stop or classify as infeasible if a required numeric QC threshold fails.
Do not interpret a nonsignificant result as no material effect without a negligible-effect interval.
Do not change canonical candidate status automatically; require governed review.
Time and cost6–12 weeks after model and reagent readiness; planning estimate only. Institution- and assay-dependent; obtain a local itemised quote before registration.
Preregistration statusdraft requires direction rationale numeric effect margin sample size and qc thresholds
Lifecycle ruleThis draft is editable. Registration requires a timestamp and checksum; a registered revision is immutable and any correction must supersede it.
Escalation ruleEscalate from L2 to L3b only after independent context-matched perturbation replication; L4 requires replicated DMD-relevant muscle evidence.
Evidence transitionCurrent evidence state → predeclared independent test → governed evidence-level review.
Data-release planRelease the frozen card, protocol identifiers, analysis code, complete denominators and results irrespective of direction; never overwrite the registered card.
Boundary: This Study Card is an evidence-gated design scaffold, not a protocol, power calculation, safety claim, prediction or therapeutic recommendation.