{"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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0001787595,0.0005218106,0.0003600914,0.0002684734,0.0001277542,0.0002626872,0.0002664698,0.0004369602,0.0006901691],"category_scores_gemma":[0.00009285248,0.0001554363,0.0002820983,0.0001351783,0.000251487,0.0001615829,0.0002519911,0.0004518552,0.0002407301],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0002817202,"about_ca_system_score_gemma":0.000210608,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0003387215,"about_ca_topic_score_gemma":0.0006953269,"domain_scores_codex":[0.999799,0.00002660175,0.00001827801,0.00005081076,0.00007226653,0.00003313478],"domain_scores_gemma":[0.9999427,0.00001013281,0.0000245434,0.000006847581,0.000006053357,0.000009747775],"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":[0.00004723923,0.00002470594,0.00006530512,0.00006269113,0.0000092148,0.00007382129,0.00001066734,0.0001919379,0.9943401,0.0002891384,0.00008781867,0.004797241],"study_design_scores_gemma":[0.00004361885,0.0005557384,0.0008139757,0.000009567681,0.00004012203,0.0003975382,0.00001055711,0.001707061,0.9889614,0.00006378434,0.007387081,0.000009609957],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9363447,0.006195249,0.04908327,0.0003310049,0.0002302293,0.0004723268,0.0007613041,0.0008234914,0.005758436],"genre_scores_gemma":[0.9654841,0.00356947,0.02391657,0.0001528288,0.00002791673,0.0003278648,0.0005491001,0.00007609521,0.005895902],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.0006901691,"threshold_uncertainty_score":0.002308846,"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."}}