{"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":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.001090331,0.0001682102,0.0003055796,0.00004253539,0.0001511892,0.000008998765,0.0001222618,0.0001372716,0.000008785367],"category_scores_gemma":[0.000287189,0.0001134725,0.00003402249,0.000111863,0.0007042707,0.00001864999,0.0002150986,0.0001162277,1.26536e-7],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00003083112,"about_ca_system_score_gemma":0.00007856559,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00001451956,"about_ca_topic_score_gemma":0.00006667655,"domain_scores_codex":[0.9981065,0.000674585,0.0004671957,0.0003757334,0.0001489254,0.0002270506],"domain_scores_gemma":[0.998876,0.0002537955,0.0002816556,0.0003332683,0.0002008329,0.0000544369],"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":[0.001317242,0.0002186641,0.03313579,0.00005339873,0.00005391398,0.000001547298,0.0001647366,0.000005595391,0.9638839,0.000008673503,0.000004595741,0.001151915],"study_design_scores_gemma":[0.00243192,0.002150507,0.05727397,0.00007351312,0.00001323731,0.0000068994,0.00008810096,0.000007783146,0.9376727,0.00005337614,0.0001063133,0.0001217089],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.994579,0.004794309,0.00001580312,0.0001168906,0.00003268367,0.0003680785,0.00007768453,0.000005725792,0.00000986122],"genre_scores_gemma":[0.9984097,0.001272995,0.0001782918,0.00004630458,0.00004690559,0.00001046424,0.000009480975,0.00001511268,0.00001071265],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.02621125,"threshold_uncertainty_score":0.4627274,"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."}}