{"id":"W2517855006","doi":"10.1016/j.nmd.2016.06.444","title":"Gene Therapy 2.0: CRISPR/Cas9 genome editing provides novel therapeutic avenues for neuromuscular disorders","year":2016,"lang":"en","type":"article","venue":"Neuromuscular Disorders","topic":"CRISPR and Genetic Engineering","field":"Biochemistry, Genetics and Molecular Biology","cited_by":2,"is_retracted":false,"has_abstract":false,"ca_institutions":"Hospital for Sick Children; University of Toronto; SickKids Foundation","funders":"","keywords":"CRISPR; Genome editing; Cas9; Utrophin; Duchenne muscular dystrophy; Biology; Gene duplication; Computational biology; Genetic enhancement; Gene; Dystrophin; Bioinformatics; Genetics","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":"codex-gemma-dda1882f352a","candidate_categories":["metaepi_narrow"],"consensus_categories":[],"category_scores_codex":[0.0002337393,0.0004670454,0.0002987682,0.0001062976,0.0002198731,0.00006032972,0.0004720969,0.000197181,0.00002057383],"category_scores_gemma":[0.0001696091,0.0003813109,0.0004816706,0.0001494355,0.0001432385,0.00001788727,0.0001149352,0.0001230919,0.000009463359],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0000168135,"about_ca_system_score_gemma":0.00005592915,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00001730511,"about_ca_topic_score_gemma":0.00005766836,"domain_scores_codex":[0.9976591,0.00007372013,0.0003619335,0.0009049372,0.000281103,0.0007192004],"domain_scores_gemma":[0.9988081,0.00005901016,0.0001197652,0.0007785068,0.00008218846,0.0001524414],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"bench_or_experimental","study_design_gemma":"not_applicable","study_design_scores_codex":[0.00007396941,0.0001405173,0.001209322,0.0000491212,0.000148285,0.000001280485,0.00007519589,0.0006695986,0.9368768,0.00003812225,0.0001377571,0.06057997],"study_design_scores_gemma":[0.006513434,0.001725549,0.03857147,0.00004954045,0.0001497414,0.00003013303,0.0001547349,0.0005700212,0.2726468,0.0009574119,0.6770627,0.001568372],"study_design_candidate":"bench_or_experimental","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.6943986,0.007173208,0.2937799,0.001953163,0.000845978,0.001466428,0.0001081586,0.0001258485,0.0001487196],"genre_scores_gemma":[0.9924535,0.004382724,0.001149491,0.0006013542,0.0006285018,0.0003108796,0.00009699351,0.000188873,0.0001876591],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.676925,"threshold_uncertainty_score":0.9998639,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.01083753495435886,"score_gpt":0.2698534123215801,"score_spread":0.2590158773672212,"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."}}