{"id":"W2561412303","doi":"10.1038/gt.2016.89","title":"Deletion of the GAA repeats from the human frataxin gene using the CRISPR-Cas9 system in YG8R-derived cells and mouse models of Friedreich ataxia","year":2016,"lang":"en","type":"article","venue":"Gene Therapy","topic":"Genetic Neurodegenerative Diseases","field":"Neuroscience","cited_by":57,"is_retracted":false,"has_abstract":false,"ca_institutions":"Université Laval; Centre hospitalier universitaire de Québec","funders":"","keywords":"Frataxin; CRISPR; Biology; Ataxia; Gene; Cas9; Genetics; Intron; Cell biology; Molecular biology; Iron-binding proteins; Neuroscience","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":[0.0003380347,0.0005819541,0.0005322884,0.0008268389,0.0004270025,0.0005073681,0.000762157,0.001197536,0.00225238],"category_scores_gemma":[0.000184933,0.0003851315,0.0006216374,0.000339821,0.0006636275,0.0003765453,0.0003730712,0.001359229,0.001127751],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0006084014,"about_ca_system_score_gemma":0.0006535,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.001709097,"about_ca_topic_score_gemma":0.003265569,"domain_scores_codex":[0.9996135,0.00004489868,0.0000644384,0.000101746,0.0001101871,0.00006518593],"domain_scores_gemma":[0.9998398,0.00001879521,0.00005996037,0.00002801434,0.0000129148,0.00004061408],"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":[0.00008000046,0.00004053917,0.00006856585,0.00002941919,0.00001150409,0.0001815495,0.0000384774,0.0001344072,0.9976122,0.0005427902,0.0001224699,0.001137994],"study_design_scores_gemma":[0.00006087895,0.0001624673,0.001034273,0.00001318332,0.00004457942,0.001031049,0.00003238237,0.001377887,0.9889503,0.0001371266,0.007142136,0.00001377641],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9566309,0.001403322,0.03081004,0.0004663996,0.0002632932,0.0004506411,0.003188318,0.001064879,0.005722314],"genre_scores_gemma":[0.9563546,0.001178894,0.02378761,0.00009358868,0.00002375887,0.0003396279,0.002506173,0.0002226891,0.01549314],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.00225238,"threshold_uncertainty_score":0.007534921,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.06619763214628806,"score_gpt":0.2733005128907394,"score_spread":0.2071028807444514,"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."}}