{"id":"W3040814595","doi":"10.1016/j.bbmt.2020.07.004","title":"Busulfan Pharmacokinetics in Adenosine Deaminase-Deficient Severe Combined Immunodeficiency Gene Therapy","year":2020,"lang":"en","type":"article","venue":"Biology of Blood and Marrow Transplantation","topic":"Virus-based gene therapy research","field":"Biochemistry, Genetics and Molecular Biology","cited_by":10,"is_retracted":false,"has_abstract":false,"ca_institutions":"Université de Montréal","funders":"National Institute of Allergy and Infectious Diseases; National Heart, Lung, and Blood Institute; U.S. Food and Drug Administration; National Institutes of Health; California Institute of Regenerative Medicine; American Society of Hematology; California Institute for Regenerative Medicine; National Human Genome Research Institute; Beckman Research Institute, City of Hope; Doris Duke Charitable Foundation","keywords":"Medicine; Adenosine deaminase; Busulfan; Adenosine deaminase deficiency; Genetic enhancement; Severe combined immunodeficiency; Pharmacokinetics; Pharmacology; Adenosine; Cancer research; Internal medicine; Gene; Chemotherapy; Genetics; Cyclophosphamide","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":[0.0004014273,0.0002372866,0.0004572841,0.0002270851,0.000169273,0.0004372479,0.0001734039,0.0002821242,0.0004295185],"category_scores_gemma":[0.0006182345,0.0001440706,0.0001225775,0.0002352315,0.0002071024,0.0003897984,0.0001046665,0.0007967178,0.0001029549],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0006018239,"about_ca_system_score_gemma":0.0004828276,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0008374438,"about_ca_topic_score_gemma":0.001613293,"domain_scores_codex":[0.99985,0.00007543513,0.000007633208,0.00001513136,0.00003517104,0.00001658685],"domain_scores_gemma":[0.9998298,0.00004955693,0.00005733829,0.00001176674,0.00001243092,0.00003910157],"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":[0.0274066,0.00117597,0.006080404,0.0001795194,0.0001747826,0.0003667771,0.0002705847,0.007549204,0.8407592,0.002084024,0.001218989,0.1127339],"study_design_scores_gemma":[0.004068473,0.07602467,0.04760366,0.00007325095,0.0006742844,0.002629443,0.000228428,0.02758941,0.8180313,0.001332713,0.0216308,0.0001135135],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9920982,0.003915717,0.002338539,0.0002468109,0.00003389139,0.00005392277,0.0001540211,0.00007431392,0.001084484],"genre_scores_gemma":[0.9970449,0.0009629562,0.001197636,0.00005769109,0.0000217282,0.00004898038,0.0001104305,0.00001904113,0.0005367774],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.0008374438,"threshold_uncertainty_score":0.004366577,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.0176138323105983,"score_gpt":0.2694376880686293,"score_spread":0.251823855758031,"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."}}