{"id":"W2889344236","doi":"10.1007/978-1-4939-8651-4_27","title":"In Vitro Evaluation of Antisense-Mediated Exon Inclusion for Spinal Muscular Atrophy","year":2018,"lang":"en","type":"article","venue":"Methods in molecular biology","topic":"Neurogenetic and Muscular Disorders Research","field":"Medicine","cited_by":2,"is_retracted":false,"has_abstract":false,"ca_institutions":"Muscular Dystrophy Canada; University of Alberta","funders":"Canadian Institutes of Health Research","keywords":"Spinal muscular atrophy; SMN1; SMA*; Antisense therapy; Morpholino; Exon; Molecular biology; Genetic enhancement; Cancer research; Exon skipping; RNA splicing; Blot; Oligonucleotide; Medicine; Biology; Locked nucleic acid; Chemistry; Alternative splicing; RNA; Pathology; Gene knockdown; Cell culture; Gene; Disease; Genetics","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":[0.004008247,0.0001329239,0.0004011742,0.0004624409,0.00004154239,0.000002107509,0.0001258876,0.000205536,0.00004666145],"category_scores_gemma":[0.002111194,0.0001201712,0.0001344036,0.000486602,0.0002566518,0.00001381189,0.0002287171,0.0001615465,0.000003094026],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00006768839,"about_ca_system_score_gemma":0.000182652,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0000694233,"about_ca_topic_score_gemma":0.00001058099,"domain_scores_codex":[0.996879,0.001738905,0.000381641,0.0003895554,0.0002846722,0.0003262484],"domain_scores_gemma":[0.9990383,0.0001206473,0.00008109902,0.0003416526,0.0003585251,0.00005977079],"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":[0.0007542429,0.0001318179,0.0005859597,0.00005994695,0.00002665568,0.00001715114,0.00009327022,0.000005488635,0.9244732,0.0001059447,0.000005645541,0.07374072],"study_design_scores_gemma":[0.004562777,0.001282666,0.00272012,0.00004163895,0.00007442517,0.00001715715,0.00003507428,0.008457015,0.9757727,0.005162427,0.00175955,0.0001144007],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.6277068,0.001389086,0.3692243,0.0003238781,0.0001212452,0.001050883,0.000003841942,0.000006290472,0.0001736402],"genre_scores_gemma":[0.6001786,0.00006579366,0.3991968,0.0003354513,0.00004727958,0.000105311,0.00004173161,0.00002126439,0.000007762327],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.07362632,"threshold_uncertainty_score":0.490044,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.06867375818795222,"score_gpt":0.5044668416205138,"score_spread":0.4357930834325616,"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."}}