{"id":"W7110944318","doi":"10.1016/j.omtn.2025.102791","title":"DUX4 reduction and muscle function improvement by subcutaneous delivery of gapmer antisense oligonucleotides","year":2025,"lang":"en","type":"article","venue":"Molecular Therapy — Nucleic Acids","topic":"Muscle Physiology and Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":3,"is_retracted":false,"has_abstract":true,"ca_institutions":"Muscular Dystrophy Canada; University of Alberta","funders":"FSH Society; Muscular Dystrophy Association","keywords":"Facioscapulohumeral muscular dystrophy; Gene knockdown; Oligonucleotide; Messenger RNA; Skeletal muscle; Muscular dystrophy; Nucleic acid; Downregulation and upregulation","routes":{"ca_aff":true,"ca_fund":false,"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":[0.0001506124,0.0005048939,0.0003336148,0.0003705847,0.0001754323,0.0002080171,0.0002557959,0.0005098158,0.002422562],"category_scores_gemma":[0.00009084336,0.0001783356,0.0003294673,0.0001553856,0.0003575716,0.0002156079,0.0002036919,0.0008144175,0.0002635266],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0002038248,"about_ca_system_score_gemma":0.000254385,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0006491508,"about_ca_topic_score_gemma":0.001322238,"domain_scores_codex":[0.999845,0.00001839322,0.00001237755,0.00004549248,0.00003822639,0.00004045197],"domain_scores_gemma":[0.9999181,0.00001033743,0.00002628053,0.000008082571,0.000008021654,0.00002929091],"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":[0.000204927,0.0000436507,0.00004441465,0.00003777901,0.0000059811,0.00004635364,0.00000969448,0.00004802195,0.9978366,0.0001282355,0.00004987462,0.001544454],"study_design_scores_gemma":[0.00005702151,0.001225787,0.001072135,0.000008161486,0.00002657217,0.0001711162,0.00002772227,0.0004513363,0.99449,0.00007018435,0.002395143,0.000004789401],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9865093,0.002362424,0.006492564,0.0002999236,0.0001907498,0.00009510382,0.0004734672,0.0003535393,0.003222998],"genre_scores_gemma":[0.9794979,0.001761553,0.005003592,0.0001200577,0.00003556404,0.0001330364,0.0005586905,0.0000661906,0.01282341],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.002422562,"threshold_uncertainty_score":0.008104324,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.00328541790141773,"score_gpt":0.2076969646162068,"score_spread":0.2044115467147891,"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."}}