{"id":"W4390260665","doi":"10.1016/j.jns.2023.121728","title":"CRISPR/CAS9 system corrects PABPN1 mutation in oculopharyngeal muscular dystrophy","year":2023,"lang":"en","type":"article","venue":"Journal of the Neurological Sciences","topic":"Neurogenetic and Muscular Disorders Research","field":"Medicine","cited_by":0,"is_retracted":false,"has_abstract":false,"ca_institutions":"McGill University; Montreal Neurological Institute and Hospital","funders":"","keywords":"Oculopharyngeal muscular dystrophy; CRISPR; Muscular dystrophy; Mutation; Genetics; Biology; Gene","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":[0.0002084247,0.0003753221,0.0004092513,0.0004188326,0.0003946304,0.0003833888,0.0004655054,0.0008387839,0.003244038],"category_scores_gemma":[0.0003990885,0.0002061394,0.0004287307,0.0001716714,0.0003472025,0.0001762961,0.0003696344,0.0009474714,0.0005888736],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000354826,"about_ca_system_score_gemma":0.0004242973,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.002866349,"about_ca_topic_score_gemma":0.003308143,"domain_scores_codex":[0.999685,0.0000263444,0.00004140104,0.00009068537,0.000109968,0.00004664607],"domain_scores_gemma":[0.9999093,0.00002529941,0.00002039379,0.00001245241,0.000009542641,0.00002303999],"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":[0.0002801015,0.0001231162,0.00144386,0.0003528332,0.00007009767,0.005704795,0.0001018918,0.001352936,0.9612132,0.001352978,0.0021352,0.02586892],"study_design_scores_gemma":[0.000182703,0.0003163124,0.01144969,0.0001119385,0.0002447076,0.01604494,0.0001353843,0.01304723,0.9111118,0.0006386961,0.04666248,0.00005415648],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9513495,0.003591901,0.02595088,0.0008507114,0.001110233,0.0002089244,0.002604597,0.001953043,0.01238027],"genre_scores_gemma":[0.9844252,0.0007901093,0.005736086,0.0001663698,0.00002102131,0.00005579443,0.0006692248,0.0001240699,0.008012186],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.003244038,"threshold_uncertainty_score":0.0108524,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.03483590015544393,"score_gpt":0.3211150866490344,"score_spread":0.2862791864935905,"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."}}