{"id":"W2527156470","doi":"10.1089/hum.2016.068","title":"Preclinical Testing of the Safety and Tolerability of Lentiviral Vector–Mediated Above-Normal Alpha-L-Iduronidase Expression in Murine and Human Hematopoietic Cells Using Toxicology and Biodistribution Good Laboratory Practice Studies","year":2016,"lang":"en","type":"article","venue":"Human Gene Therapy","topic":"Virus-based gene therapy research","field":"Biochemistry, Genetics and Molecular Biology","cited_by":41,"is_retracted":false,"has_abstract":true,"ca_institutions":"Smiths Detection (Canada)","funders":"Fondazione Telethon","keywords":"Genetic enhancement; Tolerability; Biodistribution; Viral vector; Transplantation; Stem cell; Haematopoiesis; Cancer research; Progenitor cell; Biology; Vector (molecular biology); Immunology; Medicine; Adverse effect; In vivo; Pharmacology; Gene; Internal medicine; Biotechnology; Cell biology; Genetics; Recombinant DNA","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":[0.001703713,0.0006203277,0.0004481063,0.0003713228,0.0002125063,0.0005462559,0.0004078738,0.000489073,0.001066954],"category_scores_gemma":[0.000701946,0.000211049,0.0004868576,0.0002420064,0.0006035371,0.0003437012,0.0002198035,0.001012003,0.0003178289],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0003477257,"about_ca_system_score_gemma":0.0005236269,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0007653306,"about_ca_topic_score_gemma":0.0006592168,"domain_scores_codex":[0.9994448,0.0001981236,0.00005771018,0.00009737985,0.0001309357,0.00007113478],"domain_scores_gemma":[0.9994352,0.0001671912,0.0001373399,0.00009200152,0.0001088916,0.00005927875],"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":[0.001534237,0.002317098,0.0006543881,0.0001577011,0.00003993587,0.0001275573,0.0001836193,0.001487954,0.9841615,0.0003191028,0.0002302545,0.008786758],"study_design_scores_gemma":[0.0001748471,0.04987545,0.002691669,0.00002692165,0.0001203366,0.000313781,0.00007684067,0.002225073,0.9415112,0.0001783497,0.002783274,0.00002218378],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9850042,0.001549096,0.00996222,0.00009664849,0.0000540733,0.0005896194,0.0005969476,0.0001350115,0.002012165],"genre_scores_gemma":[0.9893274,0.001566765,0.005579152,0.0001029037,0.00003026632,0.0004849158,0.0009061878,0.00004324925,0.001959091],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.001703713,"threshold_uncertainty_score":0.009010196,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.05502837291112342,"score_gpt":0.3635808446814577,"score_spread":0.3085524717703343,"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."}}