{"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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0001082098,0.0002538295,0.0001693082,0.0001292089,0.00009915981,0.0001268323,0.0001296256,0.0002138584,0.0008856969],"category_scores_gemma":[0.00007817936,0.00006062671,0.0001791572,0.00004953966,0.0001610434,0.00009588501,0.0000992815,0.0003083539,0.0001399014],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0001252632,"about_ca_system_score_gemma":0.0001164792,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0003960521,"about_ca_topic_score_gemma":0.0006263452,"domain_scores_codex":[0.9999409,0.00001153556,0.000005804211,0.00001538945,0.00001302882,0.00001335849],"domain_scores_gemma":[0.9999696,0.00001033975,0.000008092128,0.000002360214,0.000003822089,0.000005816325],"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":[0.00005563503,0.00002457285,0.00005638825,0.00002768987,0.000003425103,0.00002537638,0.000008311709,0.00009516493,0.9970843,0.0001063032,0.00003635741,0.00247641],"study_design_scores_gemma":[0.00002355203,0.0009878433,0.0005372905,0.000005283682,0.00001701475,0.0001235411,0.0000121893,0.0005200909,0.9958138,0.00003575093,0.001920661,0.000003074491],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.985916,0.002690164,0.0082677,0.0001618755,0.00009967824,0.00007170556,0.0001474403,0.0001591836,0.002486276],"genre_scores_gemma":[0.9900742,0.001575608,0.005520318,0.0001103349,0.00001753765,0.00006596951,0.0001482637,0.00002122926,0.002466475],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.0008856969,"threshold_uncertainty_score":0.002962947,"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."}}