{"id":"W3136047862","doi":"10.15252/emmm.202013228","title":"Targeted genome editing in vivo corrects a Dmd duplication restoring wild‐type dystrophin expression","year":2021,"lang":"en","type":"article","venue":"EMBO Molecular Medicine","topic":"CRISPR and Genetic Engineering","field":"Biochemistry, Genetics and Molecular Biology","cited_by":32,"is_retracted":false,"has_abstract":true,"ca_institutions":"Hospital for Sick Children; University of Toronto","funders":"Hospital for Sick Children; Duchenne Research Fund; Howard Hughes Medical Institute","keywords":"Sick child; Medical genetics; Genetics; Genome; Medicine; Library science; Biology; Pediatrics; Computer science; Gene","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":[0.0002098233,0.0004276488,0.00032158,0.0004077694,0.0001383042,0.000282695,0.0003618796,0.0005938035,0.001270823],"category_scores_gemma":[0.0001613431,0.0002031808,0.0003039287,0.0001600898,0.0003450686,0.0001502348,0.000342228,0.0005966159,0.0004144034],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0002561178,"about_ca_system_score_gemma":0.000265182,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0004327837,"about_ca_topic_score_gemma":0.0009567838,"domain_scores_codex":[0.9997761,0.00002711427,0.00002741134,0.00006796858,0.00007167037,0.00002981464],"domain_scores_gemma":[0.9998586,0.00003233585,0.00005778348,0.00001910157,0.000007665708,0.00002454185],"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":[0.00001891283,0.000008345557,0.00005095661,0.00001978243,0.000004434917,0.000099646,0.000008443006,0.000123186,0.9980951,0.0002427392,0.00003918371,0.001289298],"study_design_scores_gemma":[0.00001184735,0.000136577,0.0007697968,0.000008428089,0.00001939192,0.0006979074,0.0000111817,0.001243768,0.9924608,0.00009565066,0.00453842,0.000006241514],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.930488,0.001307398,0.06055285,0.0003829896,0.0002052151,0.0001342695,0.001162032,0.001196211,0.004571068],"genre_scores_gemma":[0.966673,0.001251584,0.02400399,0.0001191949,0.00002630104,0.0000676385,0.0007291803,0.0001530763,0.006976099],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.001270823,"threshold_uncertainty_score":0.004251361,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.006892709439411191,"score_gpt":0.2780164959799537,"score_spread":0.2711237865405425,"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."}}