{"id":"W2900871300","doi":"10.1016/j.omtm.2018.11.003","title":"Gene Transfer with AAV9-PHP.B Rescues Hearing in a Mouse Model of Usher Syndrome 3A and Transduces Hair Cells in a Non-human Primate","year":2018,"lang":"en","type":"article","venue":"Molecular Therapy — Methods & Clinical Development","topic":"Hearing, Cochlea, Tinnitus, Genetics","field":"Neuroscience","cited_by":158,"is_retracted":false,"has_abstract":true,"ca_institutions":"Canadian Nuclear Laboratories","funders":"National Institute of Neurological Disorders and Stroke; National Institutes of Health; Fondation Bertarelli; Harvard Medical School; National Institute on Deafness and Other Communication Disorders; National Eye Institute; Howard Hughes Medical Institute","keywords":"Transduction (biophysics); Transgene; Hair cell; Inner ear; Biology; Genetic enhancement; Cochlea; Viral vector; Gene; Translation (biology); Gene delivery; Green fluorescent protein; Cell biology; Capsid; Vector (molecular biology); Genetics; Messenger RNA; Neuroscience; 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":["metaepi_narrow"],"consensus_categories":[],"category_scores_codex":[0.004336535,0.0004026041,0.0008491435,0.0003024705,0.0001006731,0.00005654073,0.0004619763,0.0002515785,0.00001784803],"category_scores_gemma":[0.0001539693,0.0003519973,0.0001061679,0.0004892081,0.0005022585,0.0001001645,0.0001084711,0.0005086376,0.000005194686],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00007046318,"about_ca_system_score_gemma":0.0002854802,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00005973825,"about_ca_topic_score_gemma":0.00009197386,"domain_scores_codex":[0.9948287,0.001485497,0.001453181,0.001072034,0.0005024586,0.000658131],"domain_scores_gemma":[0.9986523,0.0003740697,0.0001179504,0.0005597659,0.00007854086,0.0002173728],"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":[0.0003251086,0.0004189542,0.001933491,0.00004962327,0.00003171295,0.00006639233,0.003648405,0.0004594166,0.9619578,0.00002837839,5.556055e-7,0.03108022],"study_design_scores_gemma":[0.002419207,0.0005956905,0.01581514,0.0001389207,0.000009579134,0.00001175805,0.00003092098,0.003353793,0.9767587,0.0003073738,0.0001238635,0.0004350522],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.8293318,0.0000844403,0.1695738,0.00008374271,0.00005637883,0.0007277897,0.000003700089,0.0000381041,0.0001002538],"genre_scores_gemma":[0.6474883,0.0002399875,0.3515024,0.0005614554,0.000008405991,0.00004826725,8.056629e-7,0.00006197101,0.00008837772],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.1819287,"threshold_uncertainty_score":0.9998932,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.1299963305844325,"score_gpt":0.434897692451616,"score_spread":0.3049013618671835,"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."}}