{"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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0001809829,0.0003800619,0.000221147,0.0002546364,0.0001328596,0.0003684658,0.0002965649,0.0003707093,0.001597922],"category_scores_gemma":[0.0001417039,0.000134124,0.0002237071,0.000165458,0.0002946336,0.0001902708,0.0001499878,0.0008150825,0.0005926416],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0004804715,"about_ca_system_score_gemma":0.0003203044,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0009400844,"about_ca_topic_score_gemma":0.001319875,"domain_scores_codex":[0.9998592,0.00001469834,0.00001058943,0.00003063792,0.00006410947,0.00002069837],"domain_scores_gemma":[0.9999028,0.00001455989,0.00004333406,0.000008012631,0.00000911949,0.00002203019],"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":[0.00006216978,0.00008777493,0.0001431177,0.00003537014,0.000004929988,0.00002804883,0.00001457548,0.00009938891,0.9959217,0.0001720404,0.0001435421,0.003287247],"study_design_scores_gemma":[0.00006995466,0.001349395,0.003927725,0.00001477199,0.00002601421,0.0009358549,0.000026227,0.001797908,0.9856952,0.00006543964,0.006081903,0.000009651132],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.983026,0.0008991887,0.01143271,0.000152842,0.00003554384,0.000186181,0.0005961384,0.0004239535,0.003247482],"genre_scores_gemma":[0.9810478,0.0008200044,0.01328658,0.00006261036,0.00001203341,0.0000988578,0.001206663,0.00007104723,0.00339436],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.001597922,"threshold_uncertainty_score":0.005345523,"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."}}