{"id":"W4389047030","doi":"10.1089/nat.2023.0038","title":"Challenges of Assessing Exon 53 Skipping of the Human <i>DMD</i> Transcript with Locked Nucleic Acid-Modified Antisense Oligonucleotides in a Mouse Model for Duchenne Muscular Dystrophy","year":2023,"lang":"en","type":"article","venue":"Nucleic Acid Therapeutics","topic":"Muscle Physiology and Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":13,"is_retracted":false,"has_abstract":true,"ca_institutions":"McGill University","funders":"National Institutes of Health; Prinses Beatrix Spierfonds","keywords":"Exon skipping; Locked nucleic acid; Duchenne muscular dystrophy; Exon; Dystrophin; Molecular biology; mdx mouse; Nucleic acid; Oligonucleotide; Muscular dystrophy; RNA; Biology; DNA; Genetics; Alternative splicing; Gene","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":[0.0006851302,0.0004267677,0.0002984773,0.0002467743,0.0001208432,0.0003855037,0.0002005422,0.0005684567,0.0007905037],"category_scores_gemma":[0.0003112819,0.0001909941,0.0002590911,0.00008505734,0.000245376,0.0002338128,0.0001584412,0.0007784119,0.0003164975],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0002299321,"about_ca_system_score_gemma":0.000210119,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0005063182,"about_ca_topic_score_gemma":0.0007779514,"domain_scores_codex":[0.9996357,0.00009078898,0.00003698394,0.00009383661,0.0001062688,0.00003641431],"domain_scores_gemma":[0.9996827,0.00009219129,0.00009362781,0.00003516908,0.00005693184,0.00003935881],"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":[0.00001764866,0.000006196425,0.00006534319,0.0000185215,0.000002322636,0.000007883136,0.000004226151,0.00003720221,0.9994158,0.00002090681,0.000006575003,0.0003975433],"study_design_scores_gemma":[0.000006411445,0.000490877,0.001494587,0.00001041103,0.00002751099,0.0001508273,0.00002193706,0.0008958978,0.9959402,0.00004573617,0.0009106672,0.000004853197],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9610326,0.00262129,0.03283521,0.0002078094,0.00006747072,0.0001204681,0.0006495589,0.0003532274,0.002112461],"genre_scores_gemma":[0.9719623,0.002713624,0.02111883,0.000189966,0.00001683249,0.0001872007,0.0007135094,0.00009718304,0.003000587],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.0007905037,"threshold_uncertainty_score":0.003623307,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.04424924329904625,"score_gpt":0.2855844536500002,"score_spread":0.241335210350954,"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."}}