{"id":"W4399760955","doi":"10.1016/j.crtox.2024.100182","title":"Comprehensive review of adverse reactions and toxicology in ASO-based therapies for Duchenne Muscular Dystrophy: From FDA-approved drugs to peptide-conjugated ASO","year":2024,"lang":"en","type":"article","venue":"Current Research in Toxicology","topic":"Muscle Physiology and Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":36,"is_retracted":false,"has_abstract":true,"ca_institutions":"Muscular Dystrophy Canada; University of Alberta","funders":"Faculty of Medicine and Dentistry, University of Alberta; Muscular Dystrophy Canada; University of Alberta; Canadian Institutes of Health Research; Women and Children's Health Research Institute; Children's Health Research Institute","keywords":"Duchenne muscular dystrophy; Medicine; Exon skipping; Clinical trial; Dystrophin; Morpholino; Bioinformatics; Adverse effect; Pharmacology; Intensive care medicine; Internal medicine; Exon; Genetics; Gene; Biology","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":[0.001011189,0.0007585265,0.001233495,0.002242505,0.0003082725,0.00080395,0.00063423,0.0009471975,0.003770648],"category_scores_gemma":[0.00121303,0.0002708748,0.001008809,0.001639359,0.0003042372,0.00112308,0.0004765865,0.001047753,0.0008811955],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0004502321,"about_ca_system_score_gemma":0.0009949812,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0007634945,"about_ca_topic_score_gemma":0.00145794,"domain_scores_codex":[0.9993896,0.0001551108,0.0001436711,0.00006998227,0.0002072375,0.00003437623],"domain_scores_gemma":[0.9989983,0.000520838,0.0002008351,0.00002370354,0.0002208908,0.00003545512],"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":[0.0002109189,0.0001593706,0.0006336712,0.05053925,0.0002722466,0.00073165,0.0002577335,0.0006140416,0.007653388,0.003173654,0.04127441,0.8944796],"study_design_scores_gemma":[0.00002986117,0.0004935867,0.001595054,0.006928323,0.0004759828,0.003044456,0.0001156811,0.0002175198,0.002790166,0.0008842727,0.9833756,0.00004960933],"study_design_candidate":"not_applicable","study_design_consensus":null,"genre_codex":"review","genre_gemma":"review","genre_scores_codex":[0.0006891544,0.9966943,0.0003906382,0.0003279364,0.000231231,0.00002058117,0.0000728115,0.00001844355,0.001554937],"genre_scores_gemma":[0.00334309,0.9939774,0.0006027452,0.0005475892,0.0003132863,0.00002834424,0.0001352027,0.000005184454,0.001047162],"genre_candidate":"review","genre_consensus":"review","teacher_disagreement_score":0.003770648,"threshold_uncertainty_score":0.01261407,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.0630958557799206,"score_gpt":0.4023557635934145,"score_spread":0.3392599078134939,"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."}}