{"id":"W2593121552","doi":"10.1182/blood.v128.22.3.3","title":"Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Mean Factor IX Activity Levels of >30% without Immunosuppression","year":2016,"lang":"en","type":"article","venue":"Blood","topic":"Virus-based gene therapy research","field":"Biochemistry, Genetics and Molecular Biology","cited_by":19,"is_retracted":false,"has_abstract":true,"ca_institutions":"St. Michael's Hospital","funders":"","keywords":"Factor IX; Capsid; Immunosuppression; Medicine; Genetic enhancement; Antibody; Immunology; Single Center; Virus; Virology; Internal medicine; Molecular biology; Gene; Biology; Genetics","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":[0.0004030413,0.0003229553,0.0004114368,0.00009061787,0.0001229215,0.00001801709,0.0004411797,0.0003850188,0.0002257297],"category_scores_gemma":[0.0002934677,0.0002369215,0.0002375254,0.0001304937,0.000162708,0.00001375471,0.0000998836,0.0001213708,0.000009687387],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00003833,"about_ca_system_score_gemma":0.0002505597,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00002126185,"about_ca_topic_score_gemma":0.0001547798,"domain_scores_codex":[0.9978685,0.0002550111,0.0003498061,0.0005512055,0.0003751926,0.0006003013],"domain_scores_gemma":[0.9986999,0.0001034863,0.0001184114,0.0006115006,0.0003028213,0.0001639004],"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":[0.00350211,0.0007438264,0.00109646,0.00003322064,0.0005096782,0.000003005204,0.0001104673,0.000001075908,0.9913979,0.000003691265,0.00005877935,0.002539751],"study_design_scores_gemma":[0.008032366,0.002927697,0.00656181,0.0000387647,0.0000768043,0.000002667211,0.00001958263,0.000009161365,0.9816003,0.00004767392,0.0003613493,0.0003218207],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9954673,0.0005144244,0.0009440061,0.0002216852,0.00006999132,0.0007708285,0.001927477,0.00003827359,0.0000460223],"genre_scores_gemma":[0.9982494,0.0002089395,0.00008760512,0.00004434179,0.0001302531,0.00007987218,0.0001400201,0.00008063256,0.0009788985],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.009797631,"threshold_uncertainty_score":0.9661377,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.02101662145472926,"score_gpt":0.2852542696577755,"score_spread":0.2642376482030462,"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."}}