{"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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.00271042,0.001050629,0.001356262,0.0002794722,0.0006473677,0.001120971,0.0006712424,0.0009330133,0.001968119],"category_scores_gemma":[0.001615337,0.0001811471,0.001374522,0.0006804483,0.0007981593,0.000414868,0.0003170221,0.001078826,0.0002279153],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.008219028,"about_ca_system_score_gemma":0.01073987,"about_ca_topic_candidate":true,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.2061844,"about_ca_topic_score_gemma":0.2774424,"domain_scores_codex":[0.998926,0.0002608259,0.00004601704,0.00009393082,0.000396869,0.0002764189],"domain_scores_gemma":[0.9988343,0.0001676805,0.0002617229,0.00006884996,0.0002733194,0.0003941968],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"randomized_trial","study_design_gemma":"observational","study_design_scores_codex":[0.7064478,0.005901834,0.02381216,0.003011639,0.009353219,0.0006172786,0.0003661112,0.0067441,0.01549442,0.00264299,0.03476273,0.1908458],"study_design_scores_gemma":[0.4525346,0.1387825,0.2991529,0.001050518,0.01825798,0.0008514915,0.0003679818,0.005430876,0.009445406,0.002213383,0.07161413,0.0002982956],"study_design_candidate":"observational","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9223241,0.02702557,0.002354131,0.004695778,0.0004808668,0.002609361,0.02278836,0.0001786352,0.01754308],"genre_scores_gemma":[0.9625107,0.009954055,0.001893683,0.002266556,0.000209129,0.00141051,0.01445442,0.00005771036,0.007243245],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.7938156,"threshold_uncertainty_score":0.4099685,"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."}}