{"id":"W2950109307","doi":"10.1016/j.ymthe.2018.08.010","title":"CRISPR-Induced Deletion with SaCas9 Restores Dystrophin Expression in Dystrophic Models In Vitro and In Vivo","year":2018,"lang":"en","type":"article","venue":"Molecular Therapy","topic":"CRISPR and Genetic Engineering","field":"Biochemistry, Genetics and Molecular Biology","cited_by":80,"is_retracted":false,"has_abstract":true,"ca_institutions":"Université Laval; Centre hospitalier universitaire de Québec","funders":"Canadian Institutes of Health Research; Mitacs","keywords":"Dystrophin; Exon; Duchenne muscular dystrophy; Frameshift mutation; Biology; CRISPR; Muscular dystrophy; Intron; Exon skipping; Molecular biology; Genetics; Gene; Alternative splicing","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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0003779156,0.0004491386,0.0003709469,0.0003639299,0.0002651995,0.0003558709,0.0003986976,0.0005827239,0.00113217],"category_scores_gemma":[0.0001931588,0.0002602499,0.0003418133,0.0001119932,0.0003575038,0.0001894201,0.0003371456,0.0007052267,0.0005906018],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0003410836,"about_ca_system_score_gemma":0.0002927372,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0009132309,"about_ca_topic_score_gemma":0.00151398,"domain_scores_codex":[0.9996277,0.00008234184,0.00005489671,0.00008180257,0.0001051777,0.00004799807],"domain_scores_gemma":[0.9997724,0.00005122696,0.00006092641,0.00004263079,0.00001621269,0.00005664616],"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":[0.00002850298,0.00001657307,0.0000338118,0.00001653635,0.000004085822,0.00004755086,0.00001220631,0.00009558267,0.9992488,0.0001044603,0.00003736887,0.0003546171],"study_design_scores_gemma":[0.00001210425,0.0001668964,0.0005390021,0.000006152298,0.00001278956,0.0002217207,0.00001356086,0.001236481,0.9953123,0.00004476628,0.002428409,0.000005696721],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9582596,0.001596522,0.03262056,0.000282258,0.0002449126,0.0001439686,0.001060901,0.0006817516,0.005109491],"genre_scores_gemma":[0.9755592,0.00104825,0.01471228,0.00007448663,0.00002136472,0.0000971713,0.001057177,0.0001701373,0.007259861],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.00113217,"threshold_uncertainty_score":0.003787458,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.00966042889023605,"score_gpt":0.271645979132171,"score_spread":0.2619855502419349,"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."}}