{"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":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.000137016,0.0001804477,0.0001646292,0.0001555926,0.00002304731,0.00001788519,0.0001097991,0.0001499616,0.000004232882],"category_scores_gemma":[0.000009485533,0.0001624238,0.00002514081,0.0001561185,0.00004711215,0.000007344092,0.00003861072,0.0001249185,6.284212e-7],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00002430566,"about_ca_system_score_gemma":0.00002689403,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0001010902,"about_ca_topic_score_gemma":0.0001972346,"domain_scores_codex":[0.9989612,0.00007207502,0.0001943532,0.0003844135,0.0001208361,0.0002671266],"domain_scores_gemma":[0.999612,0.000004626569,0.00003369963,0.0002687675,0.00002945295,0.00005150223],"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":[0.0007378415,0.00004692719,0.0006646862,0.000007328264,0.000006284856,0.0000471576,0.0001466276,0.002178491,0.9948415,0.000008100335,0.000006647605,0.001308382],"study_design_scores_gemma":[0.001720166,0.0003720389,0.003129915,0.00005234694,0.00000166975,0.00001470375,0.00004378174,0.002144014,0.991955,0.0001319179,0.0002328407,0.0002015853],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9834481,0.001402039,0.01472682,0.00005834227,0.00003424308,0.0002400427,0.0000024375,0.000008463966,0.0000795186],"genre_scores_gemma":[0.9975755,0.0002462751,0.001907178,0.0001200673,0.00005299928,0.00004465912,0.000008841392,0.00003284731,0.00001158199],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.01412746,"threshold_uncertainty_score":0.6623451,"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."}}