{"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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0002160374,0.0004541457,0.0002712293,0.0002447881,0.0001060108,0.0002347058,0.0001582975,0.0002955694,0.0004510687],"category_scores_gemma":[0.0001922753,0.0001915166,0.0003119007,0.000108127,0.0001627152,0.0001606435,0.0002537627,0.0003766111,0.0001505917],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0001817076,"about_ca_system_score_gemma":0.0001363706,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0002108191,"about_ca_topic_score_gemma":0.000419893,"domain_scores_codex":[0.9997776,0.00003321801,0.00002769847,0.0000637769,0.00006912376,0.00002865765],"domain_scores_gemma":[0.9998827,0.00002489355,0.0000486397,0.00001038444,0.000009330126,0.00002413583],"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":[0.00004543423,0.0000202494,0.00008148245,0.00002436922,0.000008084768,0.00003370308,0.000008769801,0.0002118951,0.998343,0.00005257607,0.00001706003,0.001153362],"study_design_scores_gemma":[0.00002262936,0.0007245341,0.0007931558,0.000007060544,0.00004358381,0.0001899972,0.000007623748,0.001215644,0.9953021,0.00002407868,0.001660737,0.000008895503],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9910073,0.0009179476,0.006490175,0.00004861243,0.00003294995,0.0001135845,0.0001552746,0.00008673468,0.001147462],"genre_scores_gemma":[0.9879557,0.0009895101,0.0091485,0.0001025482,0.00001379934,0.0001145257,0.0002916704,0.00003924378,0.001344508],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.0004541457,"threshold_uncertainty_score":0.001508951,"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."}}