{"id":"W2515336098","doi":"10.1016/j.nmd.2016.06.152","title":"Gene editing using CRISPR/Cas9 in neuromuscular disorders","year":2016,"lang":"en","type":"article","venue":"Neuromuscular Disorders","topic":"CRISPR and Genetic Engineering","field":"Biochemistry, Genetics and Molecular Biology","cited_by":0,"is_retracted":false,"has_abstract":false,"ca_institutions":"Hospital for Sick Children","funders":"","keywords":"CRISPR; Genome editing; Cas9; Genetics; Duchenne muscular dystrophy; Gene; Biology; Exon skipping; Missense mutation; Exon; Mutation; Dystrophin; Nemaline myopathy; Computational biology; Alternative splicing","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":[0.0003631036,0.0003899431,0.000420232,0.0005894293,0.0003693239,0.0006478594,0.0006112855,0.0007665856,0.001485033],"category_scores_gemma":[0.0003448637,0.0002407882,0.0003954931,0.0002256634,0.0004730756,0.0002986458,0.0004898732,0.00109032,0.0005675099],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0004299116,"about_ca_system_score_gemma":0.0003369452,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0007525051,"about_ca_topic_score_gemma":0.001130935,"domain_scores_codex":[0.9996952,0.00004533262,0.00004236957,0.00006319409,0.000116693,0.00003722947],"domain_scores_gemma":[0.9998366,0.00004834819,0.00003978451,0.00002883491,0.00002011267,0.00002626165],"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":[0.0001416403,0.0000517828,0.0003963858,0.0002080268,0.00004509681,0.0007778636,0.00004256008,0.001012014,0.9644493,0.004010982,0.0009196886,0.02794467],"study_design_scores_gemma":[0.00003086769,0.0001142507,0.001576621,0.00004522081,0.0000786219,0.003763396,0.00003381609,0.00378422,0.9590353,0.00155056,0.02996073,0.00002630251],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.6285684,0.03066374,0.2927499,0.003200893,0.001301384,0.0003119426,0.001994922,0.005109561,0.0360993],"genre_scores_gemma":[0.9260421,0.00716349,0.05444046,0.000311323,0.0000661177,0.00006641226,0.0005690868,0.0001906342,0.01115035],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.001485033,"threshold_uncertainty_score":0.004967868,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.006628819088863041,"score_gpt":0.2680610404189565,"score_spread":0.2614322213300935,"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."}}