{"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":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.00136981,0.0001320764,0.0003661214,0.0003294316,0.00008117939,0.00000906,0.0001100291,0.00007596902,0.0001268104],"category_scores_gemma":[0.0003560217,0.0001097731,0.0001099808,0.0002652128,0.0000441102,0.0001109152,0.0002070642,0.0001761764,0.000004672485],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0001556502,"about_ca_system_score_gemma":0.0002299006,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00007970555,"about_ca_topic_score_gemma":0.000004348061,"domain_scores_codex":[0.9967427,0.0003972047,0.0006991047,0.0001626648,0.001783388,0.0002149168],"domain_scores_gemma":[0.9984299,0.00003704991,0.0004155523,0.0001786975,0.0007971054,0.0001417155],"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":[0.0007597372,0.0007685362,0.0009753107,0.00002710562,0.0000774211,0.00001402077,0.004622446,0.000003727026,0.9655498,0.000002376733,0.0002737444,0.02692577],"study_design_scores_gemma":[0.007031495,0.002523292,0.004801541,0.0005174296,0.00008098323,0.00004878745,0.001053105,0.001336773,0.9822192,0.00006176128,0.0002350748,0.00009052758],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9973139,0.001529258,0.0001143427,0.00007884529,0.0001428737,0.0004551555,9.344134e-7,0.000004419594,0.0003602459],"genre_scores_gemma":[0.9990084,0.0001546743,0.0006311056,0.0001089973,0.00005995946,0.000005319104,0.000002165406,0.00002315076,0.000006227184],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.02683524,"threshold_uncertainty_score":0.4476418,"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."}}