{"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":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0009450871,0.00009605025,0.0002599766,0.0001435347,0.00003118801,0.000002044578,0.0002202611,0.00003023513,0.000193169],"category_scores_gemma":[0.00006809922,0.00006333068,0.00004497235,0.0001591488,0.00006481612,0.000002961927,0.00003210823,0.0002188999,1.794472e-7],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00002129598,"about_ca_system_score_gemma":0.0002297221,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00001710085,"about_ca_topic_score_gemma":0.00001792818,"domain_scores_codex":[0.9986254,0.0002058297,0.0003615436,0.0001123282,0.0005495161,0.0001453788],"domain_scores_gemma":[0.9993234,0.00002420085,0.0002341486,0.0001799666,0.0001323293,0.0001060163],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"randomized_trial","study_design_gemma":"randomized_trial","study_design_scores_codex":[0.6303971,0.001171669,0.003712762,0.000004715561,0.0001255125,0.00003141997,0.0002503165,0.0002162745,0.2218667,0.000001817463,0.001326953,0.1408947],"study_design_scores_gemma":[0.7353985,0.1922331,0.0007904846,0.00001690425,0.00001369118,0.00002771149,0.00004044435,0.000004851963,0.05786096,0.00005055051,0.01348328,0.00007949401],"study_design_candidate":"randomized_trial","study_design_consensus":"randomized_trial","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.997649,0.001111652,0.00003399579,0.0007165697,0.0001998142,0.0002175724,0.000007454439,9.338058e-7,0.00006299284],"genre_scores_gemma":[0.9981136,0.0003743082,0.00006021008,0.0002104841,0.001093067,0.000003332717,0.00002970882,0.00001461065,0.0001006715],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.1910615,"threshold_uncertainty_score":0.258255,"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."}}