{"id":"W3031070702","doi":"10.1038/s41434-020-0156-6","title":"Optimization of antisense-mediated exon skipping for Duchenne muscular dystrophy","year":2020,"lang":"en","type":"review","venue":"Gene Therapy","topic":"Muscle Physiology and Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":96,"is_retracted":false,"has_abstract":false,"ca_institutions":"University of Alberta","funders":"CIHR Skin Research Training Centre; Canadian Institutes of Health Research; Muscular Dystrophy Canada; University of Alberta; Government of Canada","keywords":"Exon skipping; Duchenne muscular dystrophy; Exon; Dystrophin; Muscular dystrophy; Medicine; Bioinformatics; Biology; Genetics; Gene; 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":["metaepi_narrow"],"consensus_categories":[],"category_scores_codex":[0.00009286729,0.0002968459,0.0007937422,0.00005451876,0.0000519209,0.000006811092,0.0002144885,0.0003927856,0.00002245912],"category_scores_gemma":[0.00005414743,0.0002614458,0.0004916691,0.000131608,0.0000645531,0.000002021501,0.00004081029,0.00008627243,0.000003797869],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000007851514,"about_ca_system_score_gemma":0.0001517032,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.000003355516,"about_ca_topic_score_gemma":4.778944e-7,"domain_scores_codex":[0.9987993,0.0001189321,0.0003689002,0.0004379804,0.0000672201,0.0002076203],"domain_scores_gemma":[0.9991793,0.00002599824,0.0003036503,0.0003486862,0.00008519562,0.00005714671],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"design_other","study_design_gemma":"not_applicable","study_design_scores_codex":[0.000430166,0.0001592166,0.000001543904,0.004074874,0.001331484,0.000003653687,0.00004519927,0.001710122,0.08623369,0.00002624534,0.000885434,0.9050984],"study_design_scores_gemma":[0.0008202989,0.0005494184,0.000001826214,0.0002321835,0.0001333868,0.000004452709,0.000006393557,0.000121073,0.003570078,0.00001719743,0.9942249,0.0003187935],"study_design_candidate":"not_applicable","study_design_consensus":null,"genre_codex":"review","genre_gemma":"review","genre_scores_codex":[0.0009612851,0.9863489,0.01147119,0.00002142018,0.0001617451,0.0008433654,0.0001479177,0.00001524555,0.00002899],"genre_scores_gemma":[0.0003251675,0.9931208,0.002454427,0.0001073229,0.0003118171,0.000120582,0.003477299,0.00006335972,0.00001923399],"genre_candidate":"review","genre_consensus":"review","teacher_disagreement_score":0.9933395,"threshold_uncertainty_score":0.9999838,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.0315453696254749,"score_gpt":0.3011200228208079,"score_spread":0.269574653195333,"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."}}