{"id":"W2784203523","doi":"10.1038/s10038-017-0400-0","title":"A novel human muscle cell model of Duchenne muscular dystrophy created by CRISPR/Cas9 and evaluation of antisense-mediated exon skipping","year":2018,"lang":"en","type":"article","venue":"Journal of Human Genetics","topic":"CRISPR and Genetic Engineering","field":"Biochemistry, Genetics and Molecular Biology","cited_by":35,"is_retracted":false,"has_abstract":false,"ca_institutions":"Muscular Dystrophy Canada; University of Alberta","funders":"","keywords":"Exon skipping; Duchenne muscular dystrophy; Exon; CRISPR; Morpholino; Genome editing; Dystrophin; RNA splicing; mdx mouse; Cas9; Biology; Muscular dystrophy; HEK 293 cells; Gene; Molecular biology; Alternative splicing; Genetics; RNA; Gene knockdown","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":[],"consensus_categories":[],"category_scores_codex":[0.0005300389,0.0001730173,0.000290101,0.000123441,0.00006978237,0.00001416675,0.0001626029,0.0001500222,0.00001433157],"category_scores_gemma":[0.00005464278,0.0001724466,0.0001105774,0.00008710711,0.0001458756,0.000005929016,0.00007001082,0.0001076979,2.875379e-7],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00001715526,"about_ca_system_score_gemma":0.00006961894,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00001207524,"about_ca_topic_score_gemma":0.0000108782,"domain_scores_codex":[0.998563,0.00005116037,0.0006192735,0.0001943229,0.0003815677,0.0001906617],"domain_scores_gemma":[0.9982391,0.000009166093,0.0004519959,0.0002647323,0.0009342379,0.0001007538],"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":[0.0000215303,0.0001347294,0.0002886789,0.00006138375,0.00007520084,8.4971e-7,0.0002227051,0.006542607,0.9920186,0.00000294698,0.0002754351,0.0003553165],"study_design_scores_gemma":[0.001952311,0.001168574,0.004056585,0.00005626869,0.0002420837,0.0000185985,0.0001316232,0.01999395,0.9720107,0.00004323889,0.0001501158,0.0001759438],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9811342,0.002562017,0.01593682,0.000009711242,0.00007074328,0.0001402904,0.00003130183,0.000002957924,0.0001119845],"genre_scores_gemma":[0.9959382,0.0002547812,0.003522114,0.00001371159,0.0001787168,0.000001510517,0.00003017015,0.00003072255,0.00003011592],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.02000791,"threshold_uncertainty_score":0.7032167,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.02245604092114683,"score_gpt":0.318720431392756,"score_spread":0.2962643904716092,"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."}}