{"id":"W2911471940","doi":"10.1016/j.omtn.2019.02.003","title":"RNA-Based Therapy Utilizing Oculopharyngeal Muscular Dystrophy Transcript Knockdown and Replacement","year":2019,"lang":"en","type":"article","venue":"Molecular Therapy — Nucleic Acids","topic":"RNA Research and Splicing","field":"Biochemistry, Genetics and Molecular Biology","cited_by":20,"is_retracted":false,"has_abstract":true,"ca_institutions":"Université de Montréal; Institut National de la Recherche Scientifique; Concordia University; Centre Hospitalier de l’Université de Montréal; McGill University; Montreal Neurological Institute and Hospital","funders":"Canadian Institutes of Health Research; Muscular Dystrophy Association","keywords":"Oculopharyngeal muscular dystrophy; Gene knockdown; RNA; microRNA; Biology; Muscular dystrophy; Messenger RNA; Molecular biology; Cancer research; Genetics; Gene","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":[0.0003844801,0.0003909889,0.0003432827,0.0001127674,0.0001602594,0.0001049416,0.0003834692,0.000229001,0.0002269966],"category_scores_gemma":[0.00001548955,0.0003477535,0.0002723892,0.0001720803,0.0001282259,0.00001337654,0.0000695484,0.0001933139,0.00003596256],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00003182068,"about_ca_system_score_gemma":0.0001014482,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0000436437,"about_ca_topic_score_gemma":0.000002778762,"domain_scores_codex":[0.9975845,0.000207925,0.0003115383,0.0008065812,0.0004497385,0.0006397447],"domain_scores_gemma":[0.9986997,0.00001202668,0.00008147722,0.0008688954,0.00009741163,0.0002404349],"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":[0.001143732,0.0001116921,0.001934184,0.0000258505,0.0001988107,0.00002981007,0.00005812859,0.00006642329,0.9769766,0.00008734001,0.0001347951,0.01923267],"study_design_scores_gemma":[0.006306488,0.002494159,0.002344272,0.00003415982,0.00001307828,0.00001626504,0.00009483678,0.0009018678,0.9142901,0.00004547271,0.07293645,0.0005227902],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9868634,0.007760419,0.002817302,0.0004062684,0.0001326358,0.0008056736,0.00001281133,0.00004604408,0.001155442],"genre_scores_gemma":[0.9943192,0.001600941,0.001078671,0.001943334,0.00008640078,0.000057672,0.00008611086,0.00009803032,0.0007296548],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.07280166,"threshold_uncertainty_score":0.9998974,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.0105686837404733,"score_gpt":0.2485942718207169,"score_spread":0.2380255880802436,"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."}}