{"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":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0003416823,0.0001269803,0.0001665772,0.00005559062,0.0001327689,0.000009563573,0.0001461626,0.00009238773,0.00001094463],"category_scores_gemma":[0.00001813898,0.00009184354,0.00008132729,0.0001147401,0.0001001474,0.000002367464,0.00002494177,0.00002808993,9.355413e-7],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00002007319,"about_ca_system_score_gemma":0.00009453251,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0000141389,"about_ca_topic_score_gemma":0.00006245145,"domain_scores_codex":[0.9990829,0.0000542528,0.0002858755,0.0002324032,0.0001464147,0.0001981958],"domain_scores_gemma":[0.9994447,0.00003690675,0.0001365677,0.0002528487,0.00009257809,0.00003634612],"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":[0.001163126,0.0004692308,0.0004302008,0.000009114269,0.000131477,2.081381e-7,0.0005027459,0.00001752826,0.9682631,0.00001005426,0.000008983918,0.02899421],"study_design_scores_gemma":[0.002231046,0.007555177,0.02458048,0.000006670148,0.000009678651,4.232451e-7,0.0000219566,0.00009874528,0.9640782,0.00001336312,0.001307933,0.00009632968],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9980275,0.000847148,0.0001783129,0.00005507992,0.00004052469,0.0007765521,0.00005724096,0.00001213506,0.000005484524],"genre_scores_gemma":[0.9972115,0.001665646,0.0001024317,0.00002737787,0.00008410573,0.0002061461,0.0003571671,0.00002643495,0.0003191641],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.02889788,"threshold_uncertainty_score":0.3745271,"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."}}