{"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":"codex-gemma-dda1882f352a","candidate_categories":["metaepi_narrow"],"consensus_categories":[],"category_scores_codex":[0.0008871881,0.0004843272,0.0009095796,0.0001846568,0.0001778761,0.00003343467,0.0002939169,0.0001454814,0.0004031612],"category_scores_gemma":[0.00001723781,0.0004252247,0.0003286985,0.0003518562,0.0003237239,0.0002419752,0.00007986939,0.0003041904,0.00002417907],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0001589494,"about_ca_system_score_gemma":0.0002789829,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0003432767,"about_ca_topic_score_gemma":0.00001466844,"domain_scores_codex":[0.9966366,0.0002175997,0.0007511455,0.0006166699,0.001097953,0.0006800759],"domain_scores_gemma":[0.9982176,0.00004389884,0.0003084855,0.0007332333,0.000366835,0.0003299824],"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":[0.002168393,0.0008647085,0.0002843049,0.00006233511,0.0005644326,0.0001416973,0.001058644,0.00001928437,0.9723583,0.00002285005,0.007424747,0.01503027],"study_design_scores_gemma":[0.01213073,0.002225347,0.000449322,0.00007471975,0.00007143999,0.0000890665,0.0001613767,0.00008447532,0.9744727,0.000445637,0.009421569,0.0003736402],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.8976082,0.09952557,0.0006549281,0.0002519105,0.0001709742,0.001279293,0.0002668602,0.00008826719,0.0001539696],"genre_scores_gemma":[0.9800423,0.01291372,0.003638096,0.000286755,0.0004571763,0.00006154221,0.0006556861,0.0001502559,0.001794495],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.08661185,"threshold_uncertainty_score":0.9998199,"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."}}