{"id":"W2074841269","doi":"10.1111/hae.12411","title":"Current status of haemophilia gene therapy","year":2014,"lang":"en","type":"review","venue":"Haemophilia","topic":"Virus-based gene therapy research","field":"Biochemistry, Genetics and Molecular Biology","cited_by":76,"is_retracted":false,"has_abstract":true,"ca_institutions":"Queen's University","funders":"Rosetrees Trust; National Institute for Health and Care Research","keywords":"Haemophilia; Genetic enhancement; Medicine; Haemophilia B; Factor IX; Transgene; Haemophilia A; Transduction (biophysics); Gene delivery; Gene transfer; Gene; Immunology; Bioinformatics; Biology; Genetics; Internal medicine; Surgery","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":["metaepi_narrow"],"consensus_categories":[],"category_scores_codex":[0.0006080106,0.0007886944,0.001924358,0.000275135,0.00009366373,0.00003362136,0.001044522,0.0006142717,0.0001735833],"category_scores_gemma":[0.0001275089,0.000661046,0.001059511,0.0003709539,0.0003038392,0.000003535738,0.0003275054,0.0005230131,0.0001695376],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00008001139,"about_ca_system_score_gemma":0.001017473,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00001621906,"about_ca_topic_score_gemma":0.00002280561,"domain_scores_codex":[0.9958067,0.0005738618,0.0009761481,0.001045617,0.0005688185,0.001028847],"domain_scores_gemma":[0.9968642,0.00005449438,0.0005534483,0.001910598,0.0002455299,0.0003717394],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"design_other","study_design_gemma":"not_applicable","study_design_scores_codex":[0.0002995953,0.0001560236,0.00000919888,0.003457839,0.0003371117,0.000005103334,0.00001318246,0.000003249295,0.002201407,0.00001772681,0.003139016,0.9903606],"study_design_scores_gemma":[0.0006122949,0.001879056,0.00000593374,0.001184228,0.000122119,0.00002232397,0.000001590264,0.000002940583,0.009082564,0.0000707755,0.986409,0.0006071761],"study_design_candidate":"design_other","study_design_consensus":null,"genre_codex":"review","genre_gemma":"review","genre_scores_codex":[0.002179688,0.9953023,0.00005253441,0.00001370646,0.0005700089,0.001211378,0.0003620837,0.00002714802,0.0002811041],"genre_scores_gemma":[0.0002567025,0.9962044,0.0001139878,0.00005339511,0.001104854,0.0001873958,0.001487876,0.0002005507,0.0003908002],"genre_candidate":"review","genre_consensus":"review","teacher_disagreement_score":0.9897534,"threshold_uncertainty_score":0.9995841,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.06755464702954414,"score_gpt":0.3856219036215462,"score_spread":0.3180672565920021,"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."}}