{"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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0008798711,0.0007409697,0.0006936861,0.001021581,0.0001592301,0.001220813,0.0004798069,0.001046746,0.004830116],"category_scores_gemma":[0.0005979571,0.0001946078,0.0005862573,0.0005765318,0.0003946653,0.001032713,0.0004542975,0.001096022,0.002215584],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0004654712,"about_ca_system_score_gemma":0.0007135417,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0003919241,"about_ca_topic_score_gemma":0.0004424613,"domain_scores_codex":[0.9996483,0.00007258937,0.00004759047,0.00007010006,0.0001272412,0.00003415124],"domain_scores_gemma":[0.9997212,0.0001241674,0.00004735286,0.00001136424,0.00007150717,0.00002437161],"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":[0.0002260786,0.0001343072,0.0004169252,0.01111065,0.0001042818,0.0003807701,0.000159195,0.0004647062,0.04358196,0.006286365,0.01800334,0.9191314],"study_design_scores_gemma":[0.00007108908,0.0007606809,0.001351435,0.00219932,0.0002432546,0.004084556,0.0001013522,0.0003312266,0.01774413,0.003769997,0.9693015,0.00004135216],"study_design_candidate":"not_applicable","study_design_consensus":null,"genre_codex":"review","genre_gemma":"review","genre_scores_codex":[0.002071754,0.9814384,0.00507895,0.0009223369,0.0004792465,0.000042755,0.0001019966,0.0001187746,0.009745829],"genre_scores_gemma":[0.01294362,0.9783337,0.002941523,0.0009466688,0.0006046827,0.00007272888,0.0003178143,0.00003119811,0.003807995],"genre_candidate":"review","genre_consensus":"review","teacher_disagreement_score":0.004830116,"threshold_uncertainty_score":0.01615834,"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."}}