{"id":"W2889393717","doi":"10.1007/978-1-4939-8651-4_5","title":"Tips to Design Effective Splice-Switching Antisense Oligonucleotides for Exon Skipping and Exon Inclusion","year":2018,"lang":"en","type":"article","venue":"Methods in molecular biology","topic":"Neurogenetic and Muscular Disorders Research","field":"Medicine","cited_by":15,"is_retracted":false,"has_abstract":false,"ca_institutions":"Muscular Dystrophy Canada; University of Alberta","funders":"Canadian Institutes of Health Research; Ministry of Education, India; Ministry of Earth Sciences","keywords":"Exon; Exon skipping; Spinal muscular atrophy; Duchenne muscular dystrophy; Muscular dystrophy; Limb-girdle muscular dystrophy; Dystrophin; mdx mouse; RNA splicing; Splice site mutation; Dysferlin; Medicine; Alternative splicing; Genetics; Biology; Mutation; Gene; RNA","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":[0.0010756,0.001184696,0.0007311761,0.0005595197,0.0005202494,0.0006565696,0.0007715204,0.001172279,0.005667532],"category_scores_gemma":[0.001518309,0.0008255867,0.0006433087,0.0004000207,0.0004426609,0.0009984073,0.0006080487,0.002714907,0.005063269],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0004398773,"about_ca_system_score_gemma":0.0006959974,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.000342406,"about_ca_topic_score_gemma":0.0008903659,"domain_scores_codex":[0.9994994,0.00007831375,0.00006368284,0.00007933791,0.0002142332,0.00006505528],"domain_scores_gemma":[0.9993663,0.0001975247,0.000115676,0.00007087514,0.0001750899,0.00007444237],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"bench_or_experimental","study_design_gemma":"not_applicable","study_design_scores_codex":[0.0001897413,0.000139062,0.0001791048,0.0007926917,0.00003042205,0.000370189,0.0001913518,0.003624629,0.9106841,0.01242505,0.005871586,0.06550214],"study_design_scores_gemma":[0.00009941064,0.0004977842,0.0001428405,0.0000878199,0.00004222913,0.0003624135,0.00007576658,0.005880277,0.937987,0.006063845,0.04870126,0.0000593372],"study_design_candidate":"not_applicable","study_design_consensus":null,"genre_codex":"methods","genre_gemma":"methods","genre_scores_codex":[0.1001937,0.007394971,0.8616987,0.002742385,0.001949234,0.001081135,0.001592222,0.004800053,0.01854767],"genre_scores_gemma":[0.2163606,0.01038348,0.7473183,0.002392667,0.0004249355,0.002165248,0.001817062,0.00103976,0.018098],"genre_candidate":"methods","genre_consensus":"methods","teacher_disagreement_score":0.005667532,"threshold_uncertainty_score":0.01895982,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.04124364104873005,"score_gpt":0.462615692825383,"score_spread":0.4213720517766529,"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."}}