Genome repair / Observational data reanalysis
See clinical-summary calculations
Clinical function does not identify edited-cell coverage
One infant, unequal sampling windows, unknown specimen counts and continuing care prevent an isolated causal effect. Liver biopsy was deferred; corrected-cell fractions and any ordinary-aging coverage threshold remain unknown.
{
"analysis_type": "Single-patient descriptive contrasts of published summaries; no raw-series reconstruction or inference",
"independent_patients": 1,
"summary_rows": [
{
"endpoint": "ammonia",
"unit": "umol/L",
"group": "baseline",
"median": 23,
"q1": 14,
"q3": 48,
"IQR_width": 34,
"n_measurements": null,
"independent_patients": 1
},
{
"endpoint": "ammonia",
"unit": "umol/L",
"group": "between",
"median": 9,
"q1": 9,
"q3": 19,
"IQR_width": 10,
"n_measurements": null,
"independent_patients": 1
},
{
"endpoint": "ammonia",
"unit": "umol/L",
"group": "after_second",
"median": 13,
"q1": 9,
"q3": 28,
"IQR_width": 19,
"n_measurements": null,
"independent_patients": 1
},
{
"endpoint": "urinary_orotic_acid",
"unit": "mmol/mol creatinine",
"group": "baseline",
"median": 1.7,
"q1": 1.6,
"q3": 1.8,
"IQR_width": 0.19999999999999996,
"n_measurements": null,
"independent_patients": 1
},
{
"endpoint": "urinary_orotic_acid",
"unit": "mmol/mol creatinine",
"group": "after_first_steady_state",
"median": 2.4,
"q1": 2.0,
"q3": 3.0,
"IQR_width": 1.0,
"n_measurements": null,
"independent_patients": 1
},
{
"endpoint": "urinary_orotic_acid",
"unit": "mmol/mol creatinine",
"group": "after_second_steady_state",
"median": 2.6,
"q1": 2.0,
"q3": 3.6,
"IQR_width": 1.6,
"n_measurements": null,
"independent_patients": 1
}
],
"contrasts": [
{
"endpoint": "ammonia",
"unit": "umol/L",
"comparison": "between_vs_baseline",
"difference_of_reported_medians": -14,
"ratio_of_reported_medians": 0.391304347826087,
"relative_difference_of_reported_medians_percent": -60.86956521739131,
"independent_patients": 1,
"interpretation": "Descriptive contrast of group summaries; not a paired-specimen ratio or causal effect"
},
{
"endpoint": "ammonia",
"unit": "umol/L",
"comparison": "after_second_vs_baseline",
"difference_of_reported_medians": -10,
"ratio_of_reported_medians": 0.5652173913043478,
"relative_difference_of_reported_medians_percent": -43.47826086956522,
"independent_patients": 1,
"interpretation": "Descriptive contrast of group summaries; not a paired-specimen ratio or causal effect"
},
{
"endpoint": "urinary_orotic_acid",
"unit": "mmol/mol creatinine",
"comparison": "after_first_steady_state_vs_baseline",
"difference_of_reported_medians": 0.7,
"ratio_of_reported_medians": 1.411764705882353,
"relative_difference_of_reported_medians_percent": 41.176470588235304,
"independent_patients": 1,
"interpretation": "Descriptive contrast of group summaries; not a paired-specimen ratio or causal effect"
},
{
"endpoint": "urinary_orotic_acid",
"unit": "mmol/mol creatinine",
"comparison": "after_second_steady_state_vs_baseline",
"difference_of_reported_medians": 0.9000000000000001,
"ratio_of_reported_medians": 1.5294117647058825,
"relative_difference_of_reported_medians_percent": 52.941176470588246,
"independent_patients": 1,
"interpretation": "Descriptive contrast of group summaries; not a paired-specimen ratio or causal effect"
}
],
"timeline": {
"time_unit": "day of life",
"second_intervention_day_derived": 230,
"first_to_followup_end_elapsed_days": 48,
"first_to_followup_end_inclusive_day_count": 49,
"second_to_followup_end_elapsed_days": 26,
"nominal_relative_medication_reduction_day_derived_from_weeks": 244,
"nominal_relative_medication_reduction_to_followup_end_days": 12,
"medication_timing_precision": "Week-based source description converted arithmetically; not a reported exact clinical day",
"reported_followup_after_first_weeks": 7,
"reported_illness_window_after_second_weeks": 4,
"rounding_note": "Rounded source week descriptions and calendar conventions are retained, not substituted for timestamp differences"
},
"weight_contrast": {
"unit": "kg",
"difference": 1.0300000000000002,
"relative_difference_percent": 14.425770308123242,
"elapsed_days": 49,
"reported_percentile_point_difference": 17,
"interpretation": "Descriptive growth in an infant; not an editing-attributable effect or percentile-to-weight conversion"
},
"reported_relative_medication_fraction_of_start": 0.5,
"unavailable_inferences": {
"raw_clinical_trajectory": "No numeric specimen series or complete specimen counts acquired",
"protein_intake_percent_change": "No numeric intake series reconstructed; age-specific feeding target changes",
"paired_ammonia_orotic_association": "Specimen correspondence and identical sampling windows unavailable",
"clinical_causal_effect": "One uncontrolled case with concurrent care, illness, growth and unequal observation windows",
"patient_corrected_allele_fraction": "No patient liver biopsy; unknown",
"patient_corrected_hepatocyte_fraction": "No patient liver biopsy; allele-to-cell mapping also unavailable",
"joint_corrected_viable_functional_cell_fraction": "No common patient molecular/cellular/function sampling frame",
"ordinary_aging_coverage_fraction": "Inherited-lesion correction does not supply a natural-aging causal target or coverage threshold",
"long_term_safety_or_durability": "Original short follow-up; no inference from absence of reported serious events"
},
"inputs_sha256_canonical_utf8_lf": "408627b1e4ec888d3f8356e65f1944b7d8c95ea07b5b4cfe0dfdb666f757a226"
}