{"id":"W2033542081","doi":"10.1016/j.ymgme.2015.01.014","title":"Disease specific therapies in leukodystrophies and leukoencephalopathies","year":2015,"lang":"en","type":"article","venue":"Molecular Genetics and Metabolism","topic":"RNA regulation and disease","field":"Biochemistry, Genetics and Molecular Biology","cited_by":55,"is_retracted":false,"has_abstract":false,"ca_institutions":"McGill University; Montreal Children's Hospital","funders":"National Center for Chronic Disease Prevention and Health Promotion; National Institute of Neurological Disorders and Stroke; National Institute of Mental Health; Novo Nordisk Fonden; National Institutes of Health; Canadian HIV Trials Network, Canadian Institutes of Health Research; Fondation du Grand défi Pierre Lavoie; Canadian Institutes of Health Research; Genome Canada","keywords":"Leukodystrophy; Medicine; Disease; Intensive care medicine; Clinical trial; Krabbe disease; Pediatrics; Pathology","routes":{"ca_aff":true,"ca_fund":true,"ca_venue":false,"about_ca":false,"invisible_to_affiliation_only":false},"retraction":null,"screen":null,"direct_labels":[],"prediction":{"model_version":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0001214085,0.0001624827,0.000143446,0.00008116468,0.00003872421,0.00006256763,0.00007894305,0.00006309148,0.000004014485],"category_scores_gemma":[0.00003201166,0.000151892,0.00003850695,0.00008242098,0.000164425,0.000005041844,0.0001028977,0.00004581218,0.000001939185],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000004035835,"about_ca_system_score_gemma":0.0000621336,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.000007238718,"about_ca_topic_score_gemma":0.000006780869,"domain_scores_codex":[0.9991189,0.00007026964,0.0001564864,0.0003212928,0.0001451906,0.0001878957],"domain_scores_gemma":[0.9993426,0.000003016003,0.0000371452,0.0002402101,0.00005833981,0.0003187329],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"bench_or_experimental","study_design_gemma":"observational","study_design_scores_codex":[0.0005520274,0.0002167724,0.07431913,0.00006355587,0.00009146857,0.0001855824,0.0008643385,0.0007608398,0.8766856,0.01497862,0.001419639,0.02986247],"study_design_scores_gemma":[0.003333386,0.0001194187,0.6043578,0.00002026425,0.00005480307,0.00002585551,0.0005534492,0.0003698467,0.02666162,0.008571053,0.3552695,0.0006630212],"study_design_candidate":"bench_or_experimental","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.879227,0.1193953,0.0006463202,0.0002759466,0.00008667935,0.0001254009,0.00001131371,0.000007461932,0.0002245549],"genre_scores_gemma":[0.9923637,0.005865133,0.00104626,0.0003375637,0.00009594439,0.00001796153,0.00002634777,0.00002028648,0.0002267837],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.8500239,"threshold_uncertainty_score":0.6193975,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.01511170734794243,"score_gpt":0.2331041448651647,"score_spread":0.2179924375172222,"validation_status":"score_only:v0-immature-baseline","note":"Baseline scores from an immature model (maturity gate not passed). Scores rank; they never assert a category."}}