{"id":"W4229060959","doi":"10.1111/hae.14530","title":"Gene therapy – are we ready now?","year":2022,"lang":"en","type":"article","venue":"Haemophilia","topic":"Virus-based gene therapy research","field":"Biochemistry, Genetics and Molecular Biology","cited_by":14,"is_retracted":false,"has_abstract":true,"ca_institutions":"Canadian Hemophilia Society","funders":"","keywords":"Genetic enhancement; Medicine; Haemophilia; Factor IX; Adeno-associated virus; Haemophilia A; Immune system; Transgene; Bioinformatics; Gene; Vector (molecular biology); Intensive care medicine; Immunology; Genetics; Biology; Pediatrics; Internal medicine; Recombinant DNA","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":[0.0003164474,0.0001653387,0.0001472243,0.00006446258,0.0002782374,0.00002024462,0.0004581207,0.00007035748,0.0008026932],"category_scores_gemma":[0.00001162661,0.0001668247,0.00009965271,0.0001700079,0.00006989999,0.000002063257,0.0002645381,0.0001982412,0.00008553946],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00003649035,"about_ca_system_score_gemma":0.0001082032,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.000008941726,"about_ca_topic_score_gemma":0.00004758298,"domain_scores_codex":[0.9985165,0.0001883379,0.0001570791,0.0004358441,0.0003403635,0.0003618998],"domain_scores_gemma":[0.9990636,0.000008797842,0.00006725969,0.0006946908,0.00006448056,0.000101199],"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":[0.001801695,0.000155196,0.0008462607,0.000005571066,0.00007681621,0.00003949997,0.00005627464,0.0002864485,0.9418398,0.00001423946,0.04317481,0.01170334],"study_design_scores_gemma":[0.0008370532,0.002126588,0.0004594847,0.000001916804,0.000003521414,0.0000366165,0.00007383389,0.00003546283,0.5880698,0.0002082272,0.4079279,0.000219582],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9891465,0.006358625,0.00002185453,0.00267472,0.0002271198,0.0002981423,0.0001304988,0.00003203423,0.00111048],"genre_scores_gemma":[0.9872359,0.003238327,0.0001013416,0.001498504,0.0004250718,0.00017645,0.0002862561,0.00006214538,0.006975973],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.3647531,"threshold_uncertainty_score":0.8788927,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.04321706537555714,"score_gpt":0.3120832957678495,"score_spread":0.2688662303922924,"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."}}