{"id":"W2933677450","doi":"10.1007/978-3-030-03095-7_16","title":"Genome Editing for Muscle Gene Therapy","year":2019,"lang":"en","type":"book-chapter","venue":"","topic":"CRISPR and Genetic Engineering","field":"Biochemistry, Genetics and Molecular Biology","cited_by":1,"is_retracted":false,"has_abstract":false,"ca_institutions":"University of Toronto; Hospital for Sick Children; SickKids Foundation","funders":"","keywords":"CRISPR; Genome editing; Zinc finger nuclease; Duchenne muscular dystrophy; Biology; Transcription activator-like effector nuclease; Genetic enhancement; Gene; Cas9; Computational biology; Dystrophin; Muscle disorder; Genetics; Bioinformatics; Medicine","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":[0.0002085287,0.0007945544,0.000588969,0.0007939867,0.0003773196,0.001146537,0.0008605906,0.0008742082,0.0371256],"category_scores_gemma":[0.0001379067,0.0002881434,0.000342238,0.0007012616,0.0004657487,0.001075296,0.000843295,0.001550722,0.01877334],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0006044037,"about_ca_system_score_gemma":0.0003594854,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.000515032,"about_ca_topic_score_gemma":0.001568428,"domain_scores_codex":[0.9998595,0.00001205288,0.000003987068,0.00002660935,0.00008447092,0.00001346795],"domain_scores_gemma":[0.9999671,0.00001384012,0.000002792176,0.000004246619,0.00000741372,0.000004680271],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"design_other","study_design_gemma":"not_applicable","study_design_scores_codex":[0.00005956158,0.00007894661,0.00003246836,0.0009370284,0.00001739882,0.0002656108,0.0001238383,0.0009000497,0.09780736,0.1066154,0.1260634,0.6670989],"study_design_scores_gemma":[0.000004265704,0.00002036416,0.00007201012,0.00012883,0.000006871893,0.0004696035,0.00001505927,0.0003066196,0.01227742,0.0102545,0.9764356,0.000008847758],"study_design_candidate":"not_applicable","study_design_consensus":null,"genre_codex":"other","genre_gemma":"review","genre_scores_codex":[0.003082201,0.1581994,0.1361821,0.00221827,0.004640567,0.0001327993,0.0008177506,0.001423043,0.6933039],"genre_scores_gemma":[0.01061772,0.07000327,0.03212404,0.001138808,0.0004304129,0.0001239941,0.0006203753,0.0004903213,0.8844511],"genre_candidate":"review","genre_consensus":null,"teacher_disagreement_score":0.0371256,"threshold_uncertainty_score":0.1241975,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.01216740210269395,"score_gpt":0.2662070261547579,"score_spread":0.254039624052064,"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."}}