{"id":"W3111331330","doi":"10.1016/j.ymthe.2020.12.007","title":"Current Clinical Applications of In Vivo Gene Therapy with AAVs","year":2020,"lang":"en","type":"review","venue":"Molecular Therapy","topic":"Virus-based gene therapy research","field":"Biochemistry, Genetics and Molecular Biology","cited_by":745,"is_retracted":false,"has_abstract":true,"ca_institutions":"Université Laval","funders":"National Eye Institute; National Heart, Lung, and Blood Institute; Pfizer Canada; Canadian Institutes of Health Research; Novartis; Amicus Therapeutics; Sarepta Therapeutics; National Institutes of Health; Pfizer","keywords":"Genetic enhancement; Medicine; Choroideremia; Spinal muscular atrophy; Bioinformatics; Disease; Pathology; Biology; Genetics; Ophthalmology; Gene; Retinal","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":["metaepi_narrow"],"consensus_categories":[],"category_scores_codex":[0.0005391619,0.0005844468,0.00149886,0.0001922054,0.00003967944,0.00002531826,0.0009670304,0.0004508509,0.00006079829],"category_scores_gemma":[0.00001770945,0.0004534099,0.0007277807,0.0006673392,0.0002267639,0.000002685363,0.000114921,0.0005847634,0.00002542462],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00003813605,"about_ca_system_score_gemma":0.0009559359,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.000006049562,"about_ca_topic_score_gemma":0.00001882807,"domain_scores_codex":[0.9964152,0.0007593858,0.0009705188,0.001002625,0.0004212283,0.000431038],"domain_scores_gemma":[0.9979157,0.00003753804,0.0004265552,0.00128885,0.0001409181,0.0001904515],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"design_other","study_design_gemma":"not_applicable","study_design_scores_codex":[0.0009570641,0.0004595894,0.00003698858,0.0004234615,0.0004868114,0.00001918422,0.00001626104,0.0000047162,0.01133346,0.00001376755,0.00009073913,0.986158],"study_design_scores_gemma":[0.001475283,0.003433202,0.000003297997,0.000402158,0.00004167904,0.00001195299,0.000002863735,0.000002337238,0.03147408,0.00002313333,0.962685,0.0004449417],"study_design_candidate":"design_other","study_design_consensus":null,"genre_codex":"review","genre_gemma":"review","genre_scores_codex":[0.002686667,0.9927468,0.001787929,0.00004501876,0.00005894184,0.002449833,0.0001005521,0.00001811177,0.0001060936],"genre_scores_gemma":[0.0002650319,0.9974149,0.0003352447,0.0001875278,0.0002809679,0.000982894,0.0003032009,0.0001760197,0.00005417334],"genre_candidate":"review","genre_consensus":"review","teacher_disagreement_score":0.985713,"threshold_uncertainty_score":0.9997917,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.0671737211929952,"score_gpt":0.4214098242799977,"score_spread":0.3542361030870025,"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."}}