{"id":"W4409183013","doi":"10.1093/nar/gkaf241","title":"AOC 1044 induces exon 44 skipping and restores dystrophin protein in preclinical models of Duchenne muscular dystrophy","year":2025,"lang":"en","type":"article","venue":"Nucleic Acids Research","topic":"Muscle Physiology and Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":12,"is_retracted":false,"has_abstract":true,"ca_institutions":"University of Alberta","funders":"","keywords":"Duchenne muscular dystrophy; Biology; Dystrophin; Exon skipping; Exon; Muscular dystrophy; Genetics; Gene; Alternative splicing","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":[0.0002700314,0.0006866048,0.0004967909,0.0008494625,0.000244871,0.000220226,0.0003863699,0.0007931364,0.001595779],"category_scores_gemma":[0.0001433166,0.0002951338,0.0002907645,0.0002264747,0.0003643012,0.0002360469,0.0001818301,0.0008203291,0.0003935338],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0004739354,"about_ca_system_score_gemma":0.00047415,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.001160902,"about_ca_topic_score_gemma":0.001817491,"domain_scores_codex":[0.9997929,0.00003697438,0.00001878899,0.00004176788,0.00005917512,0.00005046468],"domain_scores_gemma":[0.9998553,0.00001635798,0.00004972906,0.00001300904,0.00001891439,0.00004666688],"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":[0.0003317758,0.0002702509,0.0000548857,0.00004626531,0.000009661559,0.0000464258,0.00001432619,0.0001832368,0.9968609,0.0001291301,0.0001044105,0.001948809],"study_design_scores_gemma":[0.0002361192,0.006754441,0.002000632,0.00001951128,0.0000514981,0.0002218729,0.00002491335,0.001465989,0.9855728,0.0001237034,0.003516481,0.00001207142],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9908742,0.001353913,0.003645516,0.0002746253,0.00008261915,0.0001641578,0.0004621516,0.0004485985,0.002694359],"genre_scores_gemma":[0.9808549,0.002089679,0.005033156,0.0002045572,0.00002916768,0.0002659256,0.00103733,0.00007923033,0.01040619],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.001595779,"threshold_uncertainty_score":0.005338371,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.05409304834850614,"score_gpt":0.3670689343022173,"score_spread":0.3129758859537112,"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."}}