{
  "disease": "SMA",
  "records": [
    {
      "disease": "SMA",
      "candidate_or_module": "SMN1",
      "role": "etiologic_anchor",
      "establishment_basis": "human_SMA_neural_models",
      "evidence_state": "MEASURED",
      "permitted_interpretation": "Disease anchor"
    },
    {
      "disease": "SMA",
      "candidate_or_module": "SMN2",
      "role": "validated_treatment_axis",
      "establishment_basis": "source_reported_R6_MO_splicing_and_functional_correction",
      "evidence_state": "CORRECTED_SOURCE",
      "permitted_interpretation": "Positive-control treatment axis; bulk expression did not globally reverse"
    },
    {
      "disease": "SMA",
      "candidate_or_module": "KIF5A",
      "role": "downstream_candidate",
      "establishment_basis": "concordant_SMN_loss_in_two_human_neuron_contexts",
      "evidence_state": "PERTURBED",
      "permitted_interpretation": "Mechanistic candidate; source-reported functional rescue, no patient efficacy"
    },
    {
      "disease": "SMA",
      "candidate_or_module": "OXPHOS_MODULE",
      "role": "residual_program",
      "establishment_basis": "treated_patient_muscle_direction_q_gt_0.30",
      "evidence_state": "MEASURED_DIRECTIONAL",
      "permitted_interpretation": "Combination-therapy hypothesis space; no single target rank or significant module claim"
    }
  ]
}