{"id":"W2803082256","doi":"10.3791/57530","title":"Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts","year":2018,"lang":"en","type":"article","venue":"Journal of Visualized Experiments","topic":"Neurogenetic and Muscular Disorders Research","field":"Medicine","cited_by":7,"is_retracted":false,"has_abstract":true,"ca_institutions":"Muscular Dystrophy Canada; University of Alberta","funders":"Faculty of Medicine and Dentistry, University of Alberta; Muscular Dystrophy Canada; University of Alberta; Canadian Institutes of Health Research; Women and Children's Health Research Institute; Children's Health Research Institute","keywords":"Locked nucleic acid; Spinal muscular atrophy; SMA*; Oligonucleotide; Exon; Exon skipping; SMN1; Molecular biology; RNA splicing; Biology; Polymerase; Antisense therapy; Nucleic acid; Alternative splicing; DNA; Cancer research; RNA; Genetics; Gene","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":[0.0005271522,0.000363151,0.000240764,0.0001837884,0.0001700495,0.0001897722,0.000137901,0.0003391674,0.0006650045],"category_scores_gemma":[0.0002659847,0.0001380237,0.00019826,0.0001341835,0.0001857795,0.0001479147,0.0001429512,0.0003151369,0.0002149101],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0001759995,"about_ca_system_score_gemma":0.000160882,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0005429015,"about_ca_topic_score_gemma":0.0009167831,"domain_scores_codex":[0.9996337,0.0001066366,0.00005329914,0.00007485038,0.0001047104,0.00002676068],"domain_scores_gemma":[0.9997804,0.0000902107,0.00004244922,0.00002466416,0.00004353423,0.00001885382],"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":[0.00001283228,0.000008202579,0.00005762196,0.00001243744,0.000001440798,0.00003298771,0.0000158035,0.00001775058,0.9994943,0.000016045,0.000003342888,0.0003271881],"study_design_scores_gemma":[0.000003676957,0.0002052019,0.0007103711,0.000002278846,0.000009153843,0.0001095313,0.00001890398,0.000302548,0.9981791,0.0000112735,0.0004456716,0.000002298167],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.983838,0.0009128028,0.01327466,0.00005121549,0.00004516157,0.00009094275,0.0002578278,0.00009112176,0.0014382],"genre_scores_gemma":[0.9726899,0.001683317,0.02179906,0.00007463875,0.00001318121,0.0002233989,0.0005082883,0.00003975142,0.002968466],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.0006650045,"threshold_uncertainty_score":0.002787888,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.05924658317670956,"score_gpt":0.4705794578608812,"score_spread":0.4113328746841716,"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."}}