{"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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0004411909,0.000837072,0.0004280218,0.00112034,0.000362159,0.0004672984,0.000495463,0.001199319,0.00300759],"category_scores_gemma":[0.0002204276,0.0003879912,0.0006263649,0.000233476,0.0005917166,0.0004038908,0.0003557488,0.001563431,0.001061551],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000372366,"about_ca_system_score_gemma":0.0004765786,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.001609071,"about_ca_topic_score_gemma":0.002008973,"domain_scores_codex":[0.9995313,0.00006460699,0.00005456146,0.0001231456,0.0001612813,0.00006505474],"domain_scores_gemma":[0.9996843,0.00003829665,0.0000899126,0.0000381852,0.00002862794,0.0001207824],"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":[0.00005837233,0.00005122168,0.0001017377,0.00004097605,0.000008721842,0.0002514865,0.00003648373,0.00006999223,0.9972697,0.0002918708,0.0001861202,0.001633314],"study_design_scores_gemma":[0.00009334863,0.0007319154,0.003394709,0.00004991201,0.00005784745,0.002706972,0.00007129487,0.002167473,0.9791003,0.0002892243,0.01131402,0.00002302576],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9594843,0.002568413,0.02714865,0.001112455,0.0002608357,0.0001678736,0.001415827,0.001931989,0.005909717],"genre_scores_gemma":[0.9444218,0.002937087,0.02784903,0.0003508437,0.00006664027,0.0003022795,0.001606927,0.0005341743,0.02193109],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.00300759,"threshold_uncertainty_score":0.01006138,"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."}}