{"id":"W4285992907","doi":"10.1056/nejmoa2119913","title":"Phase 1–2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B","year":2022,"lang":"en","type":"article","venue":"New England Journal of Medicine","topic":"Virus-based gene therapy research","field":"Biochemistry, Genetics and Molecular Biology","cited_by":154,"is_retracted":false,"has_abstract":true,"ca_institutions":"St. Thomas Hospital","funders":"Medical Research Council; Rosetrees Trust","keywords":"Genetic enhancement; Capsid; Adeno-associated virus; Factor IX; Virology; Gene; Medicine; Virus; Internal medicine; Biology; Genetics; Recombinant DNA; Vector (molecular biology)","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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0009218301,0.001165072,0.0009891669,0.0002866059,0.000267752,0.0006666271,0.0004087118,0.0009242852,0.005187978],"category_scores_gemma":[0.000516198,0.0002829228,0.0005925594,0.0002302056,0.0005645814,0.00055698,0.0001921701,0.001974763,0.000728898],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000561173,"about_ca_system_score_gemma":0.0007700953,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0004469868,"about_ca_topic_score_gemma":0.0006384252,"domain_scores_codex":[0.9997768,0.00009183394,0.000009720669,0.00004214268,0.00002105387,0.000058383],"domain_scores_gemma":[0.999736,0.00005049975,0.00005874286,0.00002063941,0.00001520482,0.000118803],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"randomized_trial","study_design_gemma":"nonrandomized_trial","study_design_scores_codex":[0.6851058,0.07934003,0.007945687,0.0007958108,0.0009974928,0.0004799804,0.0003851032,0.002994027,0.07402798,0.0008554226,0.008391425,0.1386813],"study_design_scores_gemma":[0.6600048,0.3246769,0.00586008,0.0000270058,0.0001394856,0.0003606634,0.00003415951,0.001207536,0.004206366,0.0002482366,0.00321564,0.00001910473],"study_design_candidate":"nonrandomized_trial","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9940971,0.001372582,0.0005670903,0.0005830725,0.00009722232,0.001267364,0.0005827707,0.00008903144,0.001343886],"genre_scores_gemma":[0.9912947,0.0007621208,0.001244817,0.0007388497,0.0001698993,0.001920519,0.001496445,0.00002533788,0.002347371],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.005187978,"threshold_uncertainty_score":0.01735556,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.01790827443242138,"score_gpt":0.3073171690344181,"score_spread":0.2894088946019968,"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."}}