Current research project

DMD Minimum Perturbome

Build the first small, controlled set of DMD muscle perturbation experiments.

Candidate pool21 genes14 occupy provisional candidate slots; the rest remain replacement candidates.

Panel identities assigned24 of 2414 candidate genes plus 10 controls; still editable.

Panel slots frozen0 of 24No assignment is checksum-locked or registered yet.

Primary phenotype chosenNoSelect one main functional outcome before the study starts.

Lead Study Cards completed38%ZNF133 and MON1A drafts still have open fields.

Pilot registeredNoRegistration stays locked until the design is complete.

Do this next

Sign off the assigned panel and one primary phenotype.

All identities are provisionally assigned. Freeze the panel checksum, functional endpoint, meaningful-effect threshold and negligible-effect margin before registration.

Continue in the experiment planner

Why assigned is not the same as frozen

A target or control identity occupies a named draft panel slot. Assignment remains editable until panel freeze.

The full panel and replacement rules are checksum-locked and may change only through a versioned amendment.

Authority: PROJECTSTATE:v1.2.0-measured-dmd-evidence:DMD-MIN-PERTURBOME:1.1 · effective 2026-08-06 · source SHA-256 441d52287efd7893….

Where current evidence endsLimited muscle-context screening

What is still missingIndependent perturbation in a DMD muscle model

What this means: the platform is ready to organize a pilot, but no candidate has yet crossed the independent DMD muscle validation step.

Current project

DMD Minimum Perturbome

PROJECT-NMD-0001v1.2.0-measured-dmd-evidence

Candidate pool21

Identities assigned24 / 24

Slots frozen0 / 24

Frozen comparisons5

Draft Study Cards21

Study statusNot registered yet

Outcomes0

Open pilot Export project

Next actions

Finish the first registrable pilot

Sign off the assigned panel 24 assigned · 0 frozenVerify all candidate and control identities, then freeze the panel checksum and replacement rule.
Choose one primary phenotype MissingDefine the endpoint and minimally important effect.
Complete the two lead Study Cards In progressZNF133 Study Card: 38% · MON1A Study Card: 38%.
Register the pilot LockedFreeze donor, reagent, QC, analysis and stop rules.

Project completion0 / 4

Current state PANEL_DRAFT_ASSIGNED_SIGNOFF_PENDING. Next legal state PANEL_FROZEN_ENDPOINT_FROZEN_READY_FOR_REGISTRATION. Source project-state API.

Disease-context virtual cell

A muscle phenotype emerges from a system—not one isolated gene.

The DMD validation track must connect myotube-intrinsic responses with fibro-adipogenic, immune, vascular and extracellular-matrix context.

MyotubeFAP / stromalImmuneVascularFunction

Open the DMD validation track →

Concept visual · not an observed interaction map or experimental result.

Conceptual DMD muscle microenvironment with a central myotube, surrounding stromal, immune and vascular cells, signaling paths and abstract functional readouts.

Quick actions

Move from a gene list to an auditable object

No login · local workflow

01Search one geneOpen a typed record, evidence level, DMD state and gap badge. 02Compare a gene listRender a matrix while preserving mapping provenance and input order. 03Create a Study CardGenerate a bounded next-experiment scaffold without ranking. 04Register a state-transition testBind context, state, time, function, toxicity and replication before outcome access.

Open object registry to inspect lifecycle states, released comparison objects and draft Study Cards.

Data reality

The scientific bottleneck is visible as a funnel

Evidence gaps

21L2 observationsHepG2 perturbation observations

↓ 43%

9L3a screensExternal context, not replication

↓ 0%

0L3b replicationsIndependent myogenic perturbation absent

↓

0L4 validationsDMD functional validation absent

↓

0L5 validationsTherapeutic / clinical out of scope

Multi-fidelity path

F0–F4 shows what must happen next

F3/F4 not crossed

F0Resource17,921

Gene records and 120 SQLite tables.

F1HepG221/21

Observed same-context perturbations.

F2Context9/21

L3a screens, not replication.

×evidence break F3Independent0/21

Independent perturbation missing.

F4DMD function0/21

No current record has crossed F3 or F4.

Current scientific breakpoint: F2 → F3. The next experimental resource should produce independent disease-relevant perturbation replication before any prediction claim expands.

Capability matrix

Active tools are separated from scientifically locked tools

Prediction gate

Capability	Available?	Scientific status	Action
Gene evidence	Yes	L1–L3a evidence inspection	Explore
Study design	Yes	Draft only; prospective registration required	Create
Prediction	Infrastructure only	No prospectively validated predictions	Inspect gate
Counterfactual	No	Causal evidence absent	Requirements
Active learning	No	Calibration and acquisition budget absent	Requirements

Recent objects

Three-gene comparisonZNF133 · MON1A · GFOD2 ZNF133 Study Card 1.5Draft · registration fields open MON1A Study Card 1.5Draft · registration fields open

System status

Releasev1.2.0-measured-dmd-evidenceDOI pending

Data breadth17,921 genes120 non-empty tables

Predictions0 registeredP0 infrastructure only

External gatesPendingDOI, licence, maintainer metadata

Prediction readiness

P0PASSPrediction infrastructure only

P1LOCKED_PENDING_EXTERNAL_VALIDATIONResearch-use DMD perturbation-response prediction

P2LOCKED_NOT_IN_SCOPEClinical disease prediction or decision support

No P1 or P2 prediction claim can be made until independent disease-relevant perturbation data, prospective registration, external test results, calibration, uncertainty, OOD and abstention analyses are frozen.

Primary next asset

DMD Minimum Perturbome

A compact, well-controlled disease-relevant perturbation panel must precede any larger virtual-cell claim.

DMD and matched control myogenic systems
Two independent reagents where feasible
Imaging-first functional phenotype
Timestamped prospective holdout

Open the draft 24-slot protocol