{"id":"W2280557044","doi":"10.1016/j.gendis.2016.02.001","title":"Gene editing: A new step and a new direction toward finding a cure for Duchenne muscular dystrophy (DMD)","year":2016,"lang":"en","type":"article","venue":"Genes & Diseases","topic":"CRISPR and Genetic Engineering","field":"Biochemistry, Genetics and Molecular Biology","cited_by":0,"is_retracted":false,"has_abstract":true,"ca_institutions":"SickKids Foundation; Hospital for Sick Children; University of Toronto","funders":"National Institute of Diabetes and Digestive and Kidney Diseases; National Institutes of Health; Cystic Fibrosis Canada; Canadian Institutes of Health Research; Hospital for Sick Children; National Institute of Neurological Disorders and Stroke; Cystic Fibrosis Foundation Therapeutics","keywords":"Duchenne muscular dystrophy; Dystrophin; Exon; Muscular dystrophy; Exon skipping; Gene; Muscle disease; Disease; Neuromuscular disease; Medicine; Genome editing; Bioinformatics; Genetic enhancement; Genetics; Biology; Internal medicine; Alternative splicing; CRISPR","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":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.00003702246,0.0001799428,0.0001433221,0.00004204016,0.0000697008,0.00003383748,0.00009124174,0.00008690442,0.00001634495],"category_scores_gemma":[0.00009234336,0.0001450648,0.0001282961,0.00005261861,0.00002484524,0.000006423482,0.00007001705,0.00002180219,0.000002446565],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00001256044,"about_ca_system_score_gemma":0.00008394795,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00003448263,"about_ca_topic_score_gemma":0.00001369665,"domain_scores_codex":[0.9990986,0.00001289857,0.0001427936,0.0003831441,0.00008799989,0.0002745486],"domain_scores_gemma":[0.9994136,0.00001580192,0.0000442932,0.0002129852,0.00004176114,0.0002715681],"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":[0.00009875109,0.00002580636,0.002479164,0.00006200471,0.00009677058,0.000002921212,0.00003537169,0.00007873639,0.8019662,0.00001683649,0.008830352,0.1863071],"study_design_scores_gemma":[0.00296384,0.0005202133,0.01485668,0.0001026472,0.0003137881,0.00003639364,0.0001047609,0.000150008,0.5905434,0.0002652708,0.3895116,0.0006313709],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.738268,0.02534395,0.2346873,0.0006985737,0.0004654628,0.000343639,0.0001232778,0.00005238648,0.00001734025],"genre_scores_gemma":[0.9874254,0.001563507,0.006160734,0.00007726756,0.003208788,0.0000558532,0.00009236554,0.00004760103,0.001368459],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.3806812,"threshold_uncertainty_score":0.5915569,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.01334731338699647,"score_gpt":0.2782902035300337,"score_spread":0.2649428901430372,"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."}}