{"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":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0002655612,0.0002751031,0.0003922746,0.00009694476,0.0001520284,0.00001445476,0.0003866026,0.0002554021,0.00000189921],"category_scores_gemma":[0.00002087062,0.0002182261,0.0002222577,0.0002164053,0.000344055,0.00001739729,0.00007847599,0.0001685758,5.976748e-7],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00001439442,"about_ca_system_score_gemma":0.00007693728,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00001488421,"about_ca_topic_score_gemma":0.00008364239,"domain_scores_codex":[0.9984692,0.0001213006,0.0003980995,0.0004355254,0.000171683,0.0004041754],"domain_scores_gemma":[0.998953,0.00002382602,0.0002274414,0.0006281415,0.000121253,0.00004628958],"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":[0.0002819316,0.0001919712,0.0002226988,0.0002157333,0.0001874761,9.598981e-7,0.001091719,0.004270065,0.9906104,0.0009645846,0.000008938262,0.001953479],"study_design_scores_gemma":[0.002868618,0.0004542956,0.004261462,0.0001205799,0.0001232733,0.000002826407,0.00144447,0.01672482,0.9719865,0.001530315,0.0001133213,0.0003695024],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9965084,0.0007634101,0.001680155,0.0002738491,0.00005742364,0.0004561047,0.0000391626,0.00003296617,0.000188501],"genre_scores_gemma":[0.9983677,0.0005900869,0.0006039485,0.0002238667,0.00003740429,0.00003556194,0.0000328073,0.00007369419,0.00003488531],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.01862393,"threshold_uncertainty_score":0.8899001,"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."}}