{"id":"W4322503763","doi":"10.3390/pharmaceutics15030778","title":"Recent Trends in Antisense Therapies for Duchenne Muscular Dystrophy","year":2023,"lang":"en","type":"review","venue":"Pharmaceutics","topic":"Muscle Physiology and Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":49,"is_retracted":false,"has_abstract":true,"ca_institutions":"University of Alberta","funders":"Defeat Duchenne Canada; Canadian Institutes of Health Research; Muscular Dystrophy Canada; University of Alberta; Alberta Innovates - Health Solutions; Women and Children's Health Research Institute; Children's Health Research Institute; Alberta Innovates; U.S. Department of Defense","keywords":"Duchenne muscular dystrophy; Antisense therapy; Exon skipping; Muscular dystrophy; Medicine; Genetic enhancement; Disease; Clinical trial; Dystrophin; Drug development; Bioinformatics; Exon; Drug; Gene; Biology; Genetics; Pharmacology; Internal medicine; Alternative splicing","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":[0.0005595378,0.0008321769,0.001038876,0.00195549,0.0002170163,0.0008665184,0.0006215596,0.0009043885,0.004147661],"category_scores_gemma":[0.0006433692,0.0002793121,0.0005943842,0.001453833,0.0003387724,0.001142706,0.0006096302,0.002032725,0.002010568],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0004087071,"about_ca_system_score_gemma":0.0005941534,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0005231837,"about_ca_topic_score_gemma":0.001052328,"domain_scores_codex":[0.9998241,0.00002959047,0.00002612887,0.00003640808,0.00006579041,0.00001805833],"domain_scores_gemma":[0.999738,0.0001476536,0.00003092687,0.000005985723,0.00005585262,0.00002141325],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"design_other","study_design_gemma":"not_applicable","study_design_scores_codex":[0.0001052612,0.0001302516,0.0001396804,0.02003184,0.00009790281,0.0002788045,0.00009852286,0.0005922093,0.006246791,0.005710716,0.02640226,0.9401656],"study_design_scores_gemma":[0.00002268081,0.0001786487,0.0003834575,0.001887962,0.0001160371,0.0007225287,0.00003981373,0.0001068808,0.001037576,0.001626059,0.9938614,0.00001697069],"study_design_candidate":"not_applicable","study_design_consensus":null,"genre_codex":"review","genre_gemma":"review","genre_scores_codex":[0.0002230981,0.9977168,0.0002795653,0.000235905,0.0002341034,0.000008413927,0.00002510481,0.00001555023,0.00126148],"genre_scores_gemma":[0.0007089588,0.9978648,0.00029223,0.0002390967,0.0001264648,0.00001284002,0.0000391243,0.000002675315,0.0007139354],"genre_candidate":"review","genre_consensus":"review","teacher_disagreement_score":0.004147661,"threshold_uncertainty_score":0.01387525,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.1528171404274656,"score_gpt":0.4427749429702708,"score_spread":0.2899578025428052,"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."}}