{"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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0003706937,0.0004368542,0.0004343033,0.0004123666,0.0004624843,0.0003561465,0.0004472572,0.0008247473,0.002248951],"category_scores_gemma":[0.0001282686,0.0002175959,0.0003117286,0.0002539653,0.0003697362,0.0002547685,0.0002814149,0.000830686,0.0005738225],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0003133508,"about_ca_system_score_gemma":0.0004932158,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.002589667,"about_ca_topic_score_gemma":0.004461523,"domain_scores_codex":[0.999725,0.00003343693,0.00004073435,0.00007006706,0.00009808513,0.00003264823],"domain_scores_gemma":[0.9998518,0.00002372875,0.00003061684,0.00002626505,0.00001929187,0.00004827682],"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":[0.0001180822,0.00009483901,0.000133197,0.00003511387,0.000008493529,0.0002176172,0.00003076342,0.0001517373,0.9977393,0.0003674336,0.0001177787,0.0009855913],"study_design_scores_gemma":[0.0001063385,0.001017239,0.002184836,0.00001367054,0.000063509,0.001229194,0.00007204644,0.003086838,0.9843245,0.0001398609,0.007745991,0.00001614858],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9782339,0.000867266,0.01470092,0.0003335217,0.0002260117,0.0002370074,0.001463138,0.0002863398,0.003651929],"genre_scores_gemma":[0.9654701,0.001015724,0.01742936,0.000141753,0.00001565316,0.000345158,0.001490198,0.00005435593,0.01403761],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.002589667,"threshold_uncertainty_score":0.007523477,"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."}}