{"id":"W2515336098","doi":"10.1016/j.nmd.2016.06.152","title":"Gene editing using CRISPR/Cas9 in neuromuscular disorders","year":2016,"lang":"en","type":"article","venue":"Neuromuscular Disorders","topic":"CRISPR and Genetic Engineering","field":"Biochemistry, Genetics and Molecular Biology","cited_by":0,"is_retracted":false,"has_abstract":false,"ca_institutions":"Hospital for Sick Children","funders":"","keywords":"CRISPR; Genome editing; Cas9; Genetics; Duchenne muscular dystrophy; Gene; Biology; Exon skipping; Missense mutation; Exon; Mutation; Dystrophin; Nemaline myopathy; Computational biology; Alternative splicing","routes":{"ca_aff":true,"ca_fund":false,"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":[0.0002169424,0.0003815006,0.0002624768,0.0001718934,0.0001082259,0.00003376239,0.0003588184,0.000193331,0.00003720602],"category_scores_gemma":[0.0002357338,0.0003407544,0.0002771486,0.0002747059,0.0001104708,0.00001618042,0.0002054095,0.0001653193,0.00001475579],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00002592797,"about_ca_system_score_gemma":0.00004916835,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00008987648,"about_ca_topic_score_gemma":0.0003712588,"domain_scores_codex":[0.9976903,0.000126125,0.0003830684,0.0008198472,0.0002905024,0.000690125],"domain_scores_gemma":[0.9990032,0.00002663941,0.00008198929,0.0006994397,0.00003657764,0.0001521554],"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":[0.00003146067,0.0001116574,0.02255122,0.00004238776,0.0000482725,0.00001607437,0.00005665795,0.006251856,0.9486404,0.00002909284,0.0001868104,0.02203413],"study_design_scores_gemma":[0.01148458,0.001261912,0.1797583,0.0003027105,0.0002588099,0.00014871,0.0005521465,0.006104509,0.5465869,0.001207292,0.2483326,0.004001521],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9492493,0.001277477,0.0474708,0.0005699859,0.0006447659,0.0003575379,0.00001324089,0.00005353696,0.0003633047],"genre_scores_gemma":[0.9970902,0.001274424,0.0008494055,0.0002203491,0.0002940067,0.00003355301,0.00002451561,0.000115016,0.00009856519],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.4020535,"threshold_uncertainty_score":0.9999045,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.006628819088863041,"score_gpt":0.2680610404189565,"score_spread":0.2614322213300935,"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."}}