{"id":"W3093284546","doi":"10.1016/j.ymthe.2020.10.010","title":"DUX4 Transcript Knockdown with Antisense 2′-O-Methoxyethyl Gapmers for the Treatment of Facioscapulohumeral Muscular Dystrophy","year":2020,"lang":"en","type":"article","venue":"Molecular Therapy","topic":"Muscle Physiology and Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":34,"is_retracted":false,"has_abstract":false,"ca_institutions":"Muscular Dystrophy Canada; University of Alberta","funders":"National Institute of Neurological Disorders and Stroke; Canadian Institutes of Health Research; Alberta Innovates; Muscular Dystrophy Association; Canada Foundation for Innovation; University of Alberta; Women and Children's Health Research Institute; National Institutes of Health; FSH Society; Muscular Dystrophy Canada; Friends of FSH Research; FSHD Global Research Foundation","keywords":"Facioscapulohumeral muscular dystrophy; Gene knockdown; Muscular dystrophy; Biology; Messenger RNA; Molecular biology; In vivo; Skeletal muscle; Cell biology; In vitro; Gene; Genetics; Endocrinology","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":[],"consensus_categories":[],"category_scores_codex":[0.00005365571,0.0002495237,0.0002562715,0.00001999876,0.0001080737,0.0000104014,0.0001971348,0.0001181113,0.00002615321],"category_scores_gemma":[0.000008092811,0.0001583827,0.0003294007,0.0001009973,0.0001926126,0.000003685521,0.000008874104,0.00005093505,0.000002151939],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000005483442,"about_ca_system_score_gemma":0.00004730061,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.000023051,"about_ca_topic_score_gemma":0.000004425779,"domain_scores_codex":[0.9989463,0.0001025606,0.0001801165,0.0003975574,0.0001125438,0.0002608887],"domain_scores_gemma":[0.9993814,0.00002205246,0.00007776656,0.000367775,0.00006567867,0.00008538511],"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":[0.002487845,0.0001772825,0.0001316286,0.00001648335,0.0005713188,0.000006102869,0.0004346496,0.0006480375,0.9752206,0.00008413391,0.0001011665,0.02012072],"study_design_scores_gemma":[0.004631378,0.00686467,0.0007163566,0.000004161382,0.00008875904,0.000003751959,0.0001969848,0.0001959951,0.8938833,0.00006994635,0.093081,0.000263693],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.932955,0.008691369,0.05604609,0.001281507,0.00005358314,0.0008195748,0.00005775131,0.0000153577,0.00007972044],"genre_scores_gemma":[0.9947347,0.001835541,0.001660963,0.001361375,0.00008126574,0.000138039,0.0001039367,0.00004193311,0.00004222401],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.09297983,"threshold_uncertainty_score":0.645866,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.01900644403049084,"score_gpt":0.2494515575151511,"score_spread":0.2304451134846602,"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."}}