{"id":"W4389248161","doi":"10.1182/blood-2023-174468","title":"Childhood Treatment with Adeno-Associated Viral Gene Therapy Results in Stable FVIII Expression and Improved Bleeding Phenotype in Adult Severe Hemophilia A Dogs","year":2023,"lang":"en","type":"article","venue":"Blood","topic":"Virus-based gene therapy research","field":"Biochemistry, Genetics and Molecular Biology","cited_by":2,"is_retracted":false,"has_abstract":true,"ca_institutions":"Kingston Health Sciences Centre; Queen's University","funders":"","keywords":"Genetic enhancement; Medicine; Factor IX; Adeno-associated virus; Immunology; Internal medicine; Gastroenterology; Vector (molecular biology); Recombinant DNA; Gene; 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":[0.0002085879,0.0002039368,0.000258096,0.0002173215,0.0001425325,0.0002185444,0.0001402218,0.0002535213,0.001230624],"category_scores_gemma":[0.0001815432,0.0000647428,0.0001755304,0.0001044374,0.0002669725,0.000253082,0.0001210933,0.0003700015,0.0003151679],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0003166041,"about_ca_system_score_gemma":0.0001524091,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0005100955,"about_ca_topic_score_gemma":0.0008494584,"domain_scores_codex":[0.9998918,0.00001693651,0.00001391061,0.00002633217,0.00002878249,0.00002225734],"domain_scores_gemma":[0.9998724,0.00001734737,0.00004675736,0.00001222047,0.00001622528,0.0000350285],"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":[0.001105545,0.0004653109,0.009759885,0.0002286483,0.00004524592,0.0008543396,0.0002203446,0.0004163322,0.9571223,0.000273236,0.00110642,0.02840248],"study_design_scores_gemma":[0.0002220043,0.02734282,0.1128232,0.00009218358,0.00017916,0.01886663,0.000287095,0.001884796,0.8073083,0.0002137953,0.03073652,0.00004347651],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9939301,0.001964646,0.001908238,0.0001294067,0.00001820367,0.00003753383,0.0002690109,0.00008919902,0.001653562],"genre_scores_gemma":[0.9939476,0.001361368,0.001965252,0.00009171075,0.00001180834,0.00003616297,0.0007869868,0.00002333494,0.001775717],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.001230624,"threshold_uncertainty_score":0.004116833,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.01065283620522637,"score_gpt":0.2500932107645833,"score_spread":0.2394403745593569,"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."}}