{"id":"W3037823855","doi":"10.1073/pnas.1909649117","title":"Inhibition of <i>DUX4</i> expression with antisense LNA gapmers as a therapy for facioscapulohumeral muscular dystrophy","year":2020,"lang":"en","type":"article","venue":"Proceedings of the National Academy of Sciences","topic":"Muscle Physiology and Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":54,"is_retracted":false,"has_abstract":true,"ca_institutions":"Muscular Dystrophy Canada; University of Alberta","funders":"Canadian Institutes of Health Research; Canada Foundation for Innovation; University of Alberta; Muscular Dystrophy Canada; Friends of FSH Research; Government of Canada; Women and Children's Health Research Institute; National Institute of Arthritis and Musculoskeletal and Skin Diseases; FSH Society; FSHD Global Research Foundation","keywords":"Facioscapulohumeral muscular dystrophy; Muscular dystrophy; Biology; Locked nucleic acid; Oligonucleotide; Messenger RNA; Genetics; Molecular biology; Cell biology; Computational biology; 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":[],"consensus_categories":[],"category_scores_codex":[0.0001835819,0.00007462303,0.0001079812,0.0000287335,0.00008870993,0.000004042829,0.0002118387,0.00007346341,0.000004522699],"category_scores_gemma":[0.0001006534,0.00004709128,0.00007902128,0.0001719695,0.0005213088,0.00002270086,0.00004687086,0.00004923385,1.246063e-7],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000002040365,"about_ca_system_score_gemma":0.00002784958,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.000002015424,"about_ca_topic_score_gemma":1.637396e-8,"domain_scores_codex":[0.9992409,0.0000057229,0.0001587719,0.0002071337,0.0002972162,0.00009022163],"domain_scores_gemma":[0.9995386,0.00001379034,0.0002495677,0.000007404484,0.0001645,0.00002615914],"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":[0.0003901501,0.0000326576,0.0008359078,0.00004352533,0.0000151075,2.069857e-9,0.00009005028,0.00009148171,0.9972174,0.00073873,0.0003774047,0.0001676226],"study_design_scores_gemma":[0.0005552536,0.0005276026,0.002647182,0.00003057416,0.000005901714,0.000001114398,0.0001977986,0.00008072577,0.9930162,0.002648742,0.0002284044,0.00006051143],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9978971,0.0001516765,0.00002149005,0.00118964,0.000004678991,0.0002119007,0.00002188163,0.000002698107,0.0004989356],"genre_scores_gemma":[0.9980606,0.00007535311,0.001161743,0.0006310173,0.00004351557,0.00001307643,0.000001741049,0.000003643016,0.000009344309],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.004201171,"threshold_uncertainty_score":0.1920784,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.02396855748895025,"score_gpt":0.2785597522809589,"score_spread":0.2545911947920086,"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."}}