{"id":"W4405046397","doi":"10.1182/blood-2024-193453","title":"Use of Fidanacogene Elaparvovec, a Gene Therapy Vector, to Deliver a Stable, Fully Functional Human Factor IX Transgene for the Treatment of Hemophilia B: A Combined Analysis of Safety","year":2024,"lang":"en","type":"article","venue":"Blood","topic":"Virus-based gene therapy research","field":"Biochemistry, Genetics and Molecular Biology","cited_by":0,"is_retracted":false,"has_abstract":true,"ca_institutions":"Pfizer (Canada); University of Toronto; St. Michael's Hospital","funders":"","keywords":"Medicine; Adverse effect; Factor IX; Internal medicine; Dosing; Alanine transaminase; Genetic enhancement; Transaminase; Gastroenterology; Adeno-associated virus; Phases of clinical research; Clinical trial; Surgery; Recombinant DNA; Vector (molecular biology); Biology","routes":{"ca_aff":true,"ca_fund":false,"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":[0.001141257,0.0004770511,0.0006791601,0.0008840195,0.0002488514,0.0009253184,0.0004042768,0.0006853767,0.00227359],"category_scores_gemma":[0.0006645059,0.0001832264,0.0005696947,0.0005500393,0.0005154531,0.000498491,0.0003211419,0.001412523,0.0004135997],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0009569196,"about_ca_system_score_gemma":0.0009909171,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0009377532,"about_ca_topic_score_gemma":0.002020712,"domain_scores_codex":[0.9992815,0.0001405286,0.00004083118,0.0001411704,0.000302209,0.00009372619],"domain_scores_gemma":[0.999667,0.00006234487,0.00009411748,0.00004285284,0.00005246721,0.00008116057],"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":[0.01223826,0.005032141,0.005260526,0.000956408,0.0003894562,0.0004811076,0.0002001647,0.002652351,0.6599959,0.002723901,0.008230408,0.3018394],"study_design_scores_gemma":[0.01560277,0.1597625,0.02861061,0.0003032858,0.001405468,0.01088616,0.0001448955,0.005044446,0.6408077,0.001283791,0.1360006,0.0001476659],"study_design_candidate":"observational","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9350058,0.02877314,0.01367194,0.001172451,0.0002793171,0.002488456,0.00397689,0.0004759299,0.014156],"genre_scores_gemma":[0.9726411,0.005052059,0.01048571,0.000561738,0.00007487526,0.001284111,0.005107408,0.000100343,0.004692593],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.00227359,"threshold_uncertainty_score":0.00760591,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.04809121494100693,"score_gpt":0.3024543024984749,"score_spread":0.2543630875574679,"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."}}