{"id":"W4406255043","doi":"10.1002/ctm2.70073","title":"Lentivirus‐mediated gene therapy for Fabry disease: 5‐year End‐of‐Study results from the Canadian FACTs trial","year":2025,"lang":"en","type":"article","venue":"Clinical and Translational Medicine","topic":"Lysosomal Storage Disorders Research","field":"Medicine","cited_by":14,"is_retracted":false,"has_abstract":true,"ca_institutions":"Princess Margaret Cancer Centre; Hôpital Fleurimont; McMaster University; Juravinski Hospital; Western University; Dalhousie University; University Health Network; University of Toronto","funders":"Canadian Institutes of Health Research; Kidney Foundation of Canada; Midwest Athletes Against Childhood Cancer","keywords":"Fabry disease; Medicine; Globotriaosylceramide; Genetic enhancement; Progenitor cell; CD34; Alpha-galactosidase; Immunology; Adverse effect; Internal medicine; Stem cell; Disease; Biology; Gene","routes":{"ca_aff":true,"ca_fund":true,"ca_venue":false,"about_ca":true,"invisible_to_affiliation_only":false},"retraction":null,"screen":null,"direct_labels":[],"prediction":{"model_version":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.001145244,0.0001361021,0.0004337219,0.0001008521,0.0001801743,0.000009686302,0.0001365175,0.0001155699,0.0001596702],"category_scores_gemma":[0.002783982,0.0000795723,0.0001269828,0.0002551536,0.0006092464,0.0000320378,0.00001166819,0.000281574,0.00000434795],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00001298003,"about_ca_system_score_gemma":0.0009511047,"about_ca_topic_candidate":true,"about_ca_topic_consensus":true,"about_ca_topic_score_codex":0.0163865,"about_ca_topic_score_gemma":0.02091277,"domain_scores_codex":[0.9980152,0.0001748393,0.0007544098,0.0003676084,0.0004778704,0.0002100793],"domain_scores_gemma":[0.9949397,0.004165402,0.00007498191,0.000262168,0.0002161411,0.0003415445],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"observational","study_design_gemma":"observational","study_design_scores_codex":[0.2180937,0.001755363,0.6730708,0.00005374575,0.001101335,0.00004408499,0.001208836,0.000002406007,0.00009470877,0.001140668,0.006010776,0.09742353],"study_design_scores_gemma":[0.1366797,0.001250937,0.842171,0.00009812743,0.0001962132,2.675909e-7,0.0001518781,0.0001830797,0.0000109237,0.002687961,0.0164957,0.00007425107],"study_design_candidate":"observational","study_design_consensus":"observational","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9197472,0.003272961,0.0002064768,0.07140812,0.0004945088,0.003112661,0.0008046622,0.00001851441,0.0009348893],"genre_scores_gemma":[0.9962685,0.0003990378,0.00006457324,0.001442791,0.0006341634,0.00007028177,0.0008046635,0.00001113014,0.0003049099],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.1691002,"threshold_uncertainty_score":0.996953,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.1257432147456949,"score_gpt":0.4180600568836499,"score_spread":0.2923168421379549,"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."}}