{"id":"W2989359658","doi":"10.1182/blood-2019-124091","title":"Efficacy and Safety in 15 Hemophilia B Patients Treated with the AAV Gene Therapy Vector Fidanacogene Elaparvovec and Followed for at Least 1 Year","year":2019,"lang":"en","type":"article","venue":"Blood","topic":"Virus-based gene therapy research","field":"Biochemistry, Genetics and Molecular Biology","cited_by":37,"is_retracted":false,"has_abstract":true,"ca_institutions":"St. Michael's Hospital","funders":"","keywords":"Medicine; Dosing; Tolerability; Factor IX; Genetic enhancement; Adverse effect; Internal medicine; Pharmacokinetics; Gastroenterology; Pharmacology; Immunology; Urology; Biology; Gene","routes":{"ca_aff":true,"ca_fund":false,"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":[0.0001411694,0.0001556512,0.0001543529,0.00003039071,0.00007282545,0.00001820627,0.0001223834,0.00009086521,0.00002088154],"category_scores_gemma":[0.00001307094,0.0001040267,0.00003550999,0.00008310352,0.00006893596,0.000003083235,0.00006451257,0.00005823292,0.000006057715],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00001134262,"about_ca_system_score_gemma":0.0000348004,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00001229389,"about_ca_topic_score_gemma":0.0001951772,"domain_scores_codex":[0.9990921,0.00006449465,0.0001144433,0.0003549937,0.0001218375,0.0002521298],"domain_scores_gemma":[0.999505,0.00003012971,0.00004203631,0.0003071805,0.00005093196,0.0000646578],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"bench_or_experimental","study_design_gemma":"bench_or_experimental","study_design_scores_codex":[0.01263712,0.0003039537,0.1253416,0.0000124277,0.0001892533,0.000001526543,0.00007708136,0.00001121762,0.8591964,0.000002234283,0.0001085731,0.002118649],"study_design_scores_gemma":[0.02692369,0.006725824,0.1729257,0.00001295483,0.00003101992,0.000005497626,0.00001750342,0.00001783368,0.7894629,0.00000292721,0.003623006,0.000251166],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9973975,0.001297844,0.000002455348,0.0001305264,0.00001488372,0.001018612,0.0000618009,0.00000613403,0.00007023612],"genre_scores_gemma":[0.9985338,0.0004172249,0.00005673013,0.0001255669,0.00004997095,0.00004959983,0.0001285509,0.00003456113,0.0006039403],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.06973348,"threshold_uncertainty_score":0.4242087,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.00762021038364892,"score_gpt":0.2341076885924007,"score_spread":0.2264874782087518,"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."}}