{"id":"W2883634918","doi":"10.1101/378331","title":"CRISPR-induced deletion with SaCas9 restores dystrophin expression in dystrophic models <i>in vitro</i> and <i>in vivo</i>","year":2018,"lang":"en","type":"preprint","venue":"bioRxiv (Cold Spring Harbor Laboratory)","topic":"CRISPR and Genetic Engineering","field":"Biochemistry, Genetics and Molecular Biology","cited_by":0,"is_retracted":false,"has_abstract":true,"ca_institutions":"Université Laval; Centre hospitalier universitaire de Québec","funders":"","keywords":"Dystrophin; Exon; Duchenne muscular dystrophy; Biology; Spectrin; Muscular dystrophy; Gene; CRISPR; Genetics; Molecular biology; Intron; Cytoskeleton","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":[0.0002727372,0.0004603031,0.0003025627,0.0002441681,0.0002017961,0.0003146986,0.0003363709,0.0005683939,0.001710566],"category_scores_gemma":[0.0001570349,0.0001938047,0.000378865,0.00009198609,0.0003259503,0.0001558863,0.0002993879,0.0007038316,0.0007907267],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0002633031,"about_ca_system_score_gemma":0.0001612328,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0006324463,"about_ca_topic_score_gemma":0.0007314361,"domain_scores_codex":[0.9997473,0.00004825387,0.00004469449,0.00005600217,0.00006651931,0.00003723206],"domain_scores_gemma":[0.9997731,0.00004488418,0.00007601854,0.00003828734,0.0000146227,0.00005307708],"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":[0.0000344449,0.00001455962,0.00004258682,0.00002131984,0.000003997064,0.00005056997,0.000009154467,0.00005774049,0.9992557,0.00009926203,0.00006166641,0.000348915],"study_design_scores_gemma":[0.000009493504,0.0001238423,0.0007985805,0.00000549094,0.00001072399,0.0002104358,0.000011021,0.0009036047,0.9955131,0.00002955127,0.00237948,0.00000465233],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9541427,0.00126405,0.03444266,0.00034802,0.0002910144,0.0001901486,0.002367694,0.001240881,0.005712818],"genre_scores_gemma":[0.9812676,0.0005330555,0.01045781,0.0000908408,0.00001973428,0.00009372181,0.001316718,0.0002323379,0.005988228],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.001710566,"threshold_uncertainty_score":0.005722404,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.009160815768576594,"score_gpt":0.2337501678570962,"score_spread":0.2245893520885195,"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."}}