{"id":"W2966512620","doi":"10.1016/j.ymthe.2019.07.012","title":"Exons 45–55 Skipping Using Mutation-Tailored Cocktails of Antisense Morpholinos in the DMD Gene","year":2019,"lang":"en","type":"article","venue":"Molecular Therapy","topic":"Muscle Physiology and Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":45,"is_retracted":false,"has_abstract":true,"ca_institutions":"Muscular Dystrophy Canada; University of Alberta","funders":"Faculty of Medicine and Dentistry, University of Alberta; Canadian Institutes of Health Research; Hokuto Foundation for Bioscience; Muscular Dystrophy Canada; Canada Foundation for Innovation; University of Alberta; Japan Society for the Promotion of Science; Women and Children's Health Research Institute; Children's Health Research Institute","keywords":"Exon skipping; Morpholino; Dystrophin; Exon; Duchenne muscular dystrophy; Biology; Muscular dystrophy; Mutation; Molecular biology; Genetics; Cancer research; Gene; Gene knockdown; 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":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0001886235,0.0001426526,0.00016804,0.000056419,0.00004500743,0.00001060273,0.0002167369,0.0001328701,0.00003966818],"category_scores_gemma":[0.00002339639,0.000117643,0.0001161108,0.0001443534,0.00008410244,0.000004370929,0.00003125783,0.00009289002,0.00001213865],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000005568492,"about_ca_system_score_gemma":0.0000568875,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00005191058,"about_ca_topic_score_gemma":0.000006480657,"domain_scores_codex":[0.9989913,0.0001983835,0.0002131895,0.0002667221,0.0001162911,0.0002140817],"domain_scores_gemma":[0.9993706,0.00001975426,0.00009787223,0.0004335881,0.00005236633,0.00002578943],"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":[0.0001671258,0.00009063551,0.00194074,0.000009122575,0.00005237961,0.00001516439,0.0001985151,0.0025274,0.9940216,0.0001090454,0.0000266529,0.0008416202],"study_design_scores_gemma":[0.001706962,0.0002402922,0.009215628,0.00001421921,0.00001045552,0.00002511283,0.0003028954,0.0005292579,0.9867355,0.0004643264,0.0005477346,0.0002076708],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9955941,0.001622206,0.001942239,0.00009207928,0.00007733115,0.0003666655,0.000006501072,0.000005124774,0.0002937632],"genre_scores_gemma":[0.9982142,0.0001976896,0.0004047288,0.001016421,0.00002887984,0.0000133262,0.00008261598,0.00001940785,0.00002272051],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.007286153,"threshold_uncertainty_score":0.4797344,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.014146665229264,"score_gpt":0.2657224287196184,"score_spread":0.2515757634903544,"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."}}