{"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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0002200585,0.0004038854,0.000473473,0.0002269031,0.0002755228,0.0001741749,0.0003754534,0.0004782772,0.001736699],"category_scores_gemma":[0.0000887296,0.0001789882,0.0002683285,0.0001559702,0.0003620991,0.0002550582,0.0002150823,0.0007257105,0.0002171962],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0002490121,"about_ca_system_score_gemma":0.0003528979,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0009956463,"about_ca_topic_score_gemma":0.001642874,"domain_scores_codex":[0.9998176,0.00002947413,0.0000173412,0.00004208138,0.00003880894,0.0000548163],"domain_scores_gemma":[0.9999582,0.000008775004,0.000009731802,0.000005072334,0.000005064841,0.00001319277],"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":[0.0003428799,0.00009016241,0.00005559822,0.00008036858,0.00001272323,0.00005928625,0.0000224821,0.0001204552,0.9957122,0.0002565655,0.0001540142,0.00309333],"study_design_scores_gemma":[0.0001414064,0.001303445,0.001476372,0.00001055807,0.00005382329,0.0002307918,0.00003966785,0.0007368642,0.9906279,0.0000808961,0.005289183,0.000009155689],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9890541,0.003458146,0.00363512,0.000274613,0.0002259459,0.00008124415,0.0002590853,0.0002228759,0.002788858],"genre_scores_gemma":[0.9903452,0.001661096,0.002746299,0.0001170462,0.00002350616,0.000127498,0.0002500983,0.00003017387,0.004699097],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.001736699,"threshold_uncertainty_score":0.005809844,"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."}}