{"id":"W1932819137","doi":"10.1089/hum.2015.078","title":"Prevention of Lethal Murine Hypophosphatasia by Neonatal <i>Ex Vivo</i> Gene Therapy Using Lentivirally Transduced Bone Marrow Cells","year":2015,"lang":"en","type":"article","venue":"Human Gene Therapy","topic":"Alkaline Phosphatase Research Studies","field":"Medicine","cited_by":31,"is_retracted":false,"has_abstract":true,"ca_institutions":"","funders":"National Institute of Dental and Craniofacial Research; Japan Society for the Promotion of Science; Nihon University","keywords":"Hypophosphatasia; Genetic enhancement; Enzyme replacement therapy; Bone marrow; Ex vivo; Alkaline phosphatase; Medicine; Knockout mouse; Transplantation; In vivo; Internal medicine; Biology; Disease; Gene; Enzyme; Genetics; Biochemistry; Receptor","routes":{"ca_aff":false,"ca_fund":false,"ca_venue":false,"about_ca":true,"invisible_to_affiliation_only":true},"retraction":null,"screen":null,"direct_labels":[],"prediction":{"model_version":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0003366255,0.0006641709,0.0005368323,0.0004344261,0.0001954808,0.0004307776,0.0004989457,0.0007135097,0.001322803],"category_scores_gemma":[0.0001244732,0.0002071812,0.0005247566,0.000198747,0.0003897333,0.0003818292,0.000242171,0.001464524,0.0005035964],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000362846,"about_ca_system_score_gemma":0.0004225973,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0009008521,"about_ca_topic_score_gemma":0.0009913505,"domain_scores_codex":[0.9996623,0.0000433409,0.00004499166,0.00008202053,0.00009386928,0.00007346617],"domain_scores_gemma":[0.9998271,0.00001917166,0.00006220476,0.00001524733,0.00002116009,0.00005505006],"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":[0.000166611,0.000113284,0.00009331194,0.00006721533,0.000008327882,0.00005307403,0.00001963913,0.0000920987,0.9975479,0.000193192,0.00008649306,0.001558904],"study_design_scores_gemma":[0.00005651446,0.000776479,0.0008101594,0.00001241909,0.00002740589,0.000182887,0.00002096424,0.000833125,0.994844,0.00003663859,0.002393753,0.000005627027],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9724296,0.004788902,0.01623421,0.0003865592,0.0002602972,0.0002774947,0.001522185,0.0006448017,0.003455915],"genre_scores_gemma":[0.9777394,0.003708585,0.009439426,0.0002522059,0.00004994003,0.0003627462,0.00174081,0.0001242591,0.006582768],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.001322803,"threshold_uncertainty_score":0.004425228,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.0842771756065015,"score_gpt":0.3429510617180389,"score_spread":0.2586738861115374,"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."}}