{"id":"W2781785051","doi":"10.3390/biomedicines6010001","title":"Skipping Multiple Exons to Treat DMD—Promises and Challenges","year":2018,"lang":"en","type":"review","venue":"Biomedicines","topic":"CRISPR and Genetic Engineering","field":"Biochemistry, Genetics and Molecular Biology","cited_by":99,"is_retracted":false,"has_abstract":true,"ca_institutions":"Muscular Dystrophy Canada; University of Alberta","funders":"Faculty of Medicine and Dentistry, University of Alberta; University of Alberta; Women and Children's Health Research Institute; Children's Health Research Institute; Muscular Dystrophy Canada; Canadian Institutes of Health Research; Parent Project Muscular Dystrophy","keywords":"Exon skipping; Exon; Duchenne muscular dystrophy; Medicine; Bioinformatics; Muscular dystrophy; Computational biology; Pharmacology; Biology; Genetics; Gene; Internal medicine; 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":[0.000796289,0.0007061011,0.0008991975,0.001882752,0.0002625633,0.0008047394,0.000852245,0.00114352,0.002577514],"category_scores_gemma":[0.0008362035,0.0002343232,0.0004751785,0.001155353,0.0007276603,0.001615991,0.0005956262,0.002785425,0.00170241],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0006151232,"about_ca_system_score_gemma":0.0008056003,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0008020183,"about_ca_topic_score_gemma":0.001855451,"domain_scores_codex":[0.9997813,0.00003720152,0.00003248471,0.00003708066,0.00009132275,0.0000205511],"domain_scores_gemma":[0.9996074,0.0002070178,0.00004005674,0.00001259881,0.00009899892,0.00003394482],"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":[0.00005120983,0.00007720995,0.0001495905,0.009861243,0.00006544062,0.0003003478,0.00009567775,0.000376852,0.002831909,0.01112655,0.06151688,0.9135472],"study_design_scores_gemma":[0.000009545743,0.0000578315,0.0002223527,0.001505498,0.00003539274,0.001063552,0.00003529927,0.00005937539,0.0004577849,0.002275456,0.9942674,0.00001051122],"study_design_candidate":"not_applicable","study_design_consensus":null,"genre_codex":"review","genre_gemma":"review","genre_scores_codex":[0.0001066905,0.9977024,0.00018506,0.0007268313,0.0003541186,0.000002812993,0.00001276765,0.0000100774,0.0008991943],"genre_scores_gemma":[0.0007326693,0.9975191,0.0002312236,0.000534103,0.0002142719,0.000005162094,0.00002362711,0.000003248298,0.0007365783],"genre_candidate":"review","genre_consensus":"review","teacher_disagreement_score":0.002577514,"threshold_uncertainty_score":0.008622646,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.06896496417085068,"score_gpt":0.3780366884314863,"score_spread":0.3090717242606356,"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."}}