{"id":"W4386002390","doi":"10.1089/hum.2023.075","title":"Preclinical Development and Characterization of Novel Adeno-Associated Viral Vectors for the Treatment of Lipoprotein Lipase Deficiency","year":2023,"lang":"en","type":"article","venue":"Human Gene Therapy","topic":"Virus-based gene therapy research","field":"Biochemistry, Genetics and Molecular Biology","cited_by":12,"is_retracted":false,"has_abstract":true,"ca_institutions":"National Research Council Canada; BC Children's Hospital; McGill University; University of British Columbia","funders":"Canadian Institutes of Health Research","keywords":"Genetic enhancement; Biodistribution; Capsid; Adeno-associated virus; Lipoprotein lipase; Viral vector; Gene delivery; Transgene; Medicine; Pharmacology; Enzyme replacement therapy; Vector (molecular biology); Virology; Biology; Virus; Internal medicine; In vivo; Gene; Recombinant DNA; Adipose tissue; Biochemistry","routes":{"ca_aff":true,"ca_fund":true,"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":[0.000763337,0.0007389868,0.0005434438,0.0004749908,0.0002465347,0.0005281543,0.0005164135,0.000598243,0.001503526],"category_scores_gemma":[0.0003420801,0.0002831284,0.0004445039,0.0002071919,0.0003978497,0.0004951915,0.000224855,0.001450678,0.0007849744],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0005500493,"about_ca_system_score_gemma":0.0005833766,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.000658608,"about_ca_topic_score_gemma":0.0007079394,"domain_scores_codex":[0.9995561,0.00007029012,0.0000527951,0.00007820042,0.0001527119,0.00008981434],"domain_scores_gemma":[0.9997472,0.0000395237,0.00006404424,0.00003198762,0.00004960991,0.00006769429],"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":[0.0004204903,0.0009487085,0.000174358,0.0001541936,0.00001980145,0.00009080442,0.00005002726,0.0007379546,0.9899857,0.0003778834,0.0002334094,0.006806726],"study_design_scores_gemma":[0.000277125,0.01408013,0.001467524,0.00003433222,0.00007794696,0.0004343562,0.00003644309,0.002828463,0.9702953,0.0001734768,0.01027703,0.00001786666],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9516858,0.004991827,0.03498752,0.0003755069,0.0002259948,0.001909651,0.001705152,0.0004097592,0.00370882],"genre_scores_gemma":[0.945917,0.006563732,0.03297307,0.0001594131,0.00009201618,0.00221389,0.003870881,0.0001033178,0.00810665],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.001503526,"threshold_uncertainty_score":0.005029798,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.07941936829984136,"score_gpt":0.3534110679325034,"score_spread":0.273991699632662,"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."}}