{"id":"W2340356391","doi":"10.1038/gt.2016.36","title":"Development of an AAV9 coding for a 3XFLAG-TALEfrat#8-VP64 able to increase in vivo the human frataxin in YG8R mice","year":2016,"lang":"en","type":"article","venue":"Gene Therapy","topic":"CRISPR and Genetic Engineering","field":"Biochemistry, Genetics and Molecular Biology","cited_by":19,"is_retracted":false,"has_abstract":true,"ca_institutions":"Université Laval; Centre hospitalier universitaire de Québec","funders":"","keywords":"Biology; Frataxin; In vivo; Transcription (linguistics); Molecular biology; Gene; Effector; Haploinsufficiency; Gene expression; Messenger RNA; Activator (genetics); Transcription activator-like effector nuclease; Cell biology; Genetics; Genome editing; CRISPR; Iron-binding proteins","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":[],"consensus_categories":[],"category_scores_codex":[0.0003390171,0.0001159319,0.0001233239,0.00005745529,0.00004267466,0.000007901267,0.0002040509,0.00006636821,0.00001788126],"category_scores_gemma":[0.00001772869,0.00007984578,0.00003148439,0.00008229398,0.00001817921,0.000003161031,0.00004213916,0.00002938855,0.000001241838],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00001910112,"about_ca_system_score_gemma":0.00005323085,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00006113628,"about_ca_topic_score_gemma":0.0008807732,"domain_scores_codex":[0.999235,0.00002669749,0.0002267249,0.0002251706,0.00006654947,0.0002198807],"domain_scores_gemma":[0.9996161,0.00001500165,0.00003471203,0.000253844,0.00002934888,0.00005098302],"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":[0.0001045344,0.00004858828,0.0004909132,0.000007322664,0.00001281019,6.885909e-7,0.0004581963,0.0001462664,0.989141,0.00001617403,0.00009875485,0.009474725],"study_design_scores_gemma":[0.001059566,0.000148834,0.003142896,0.00002381035,0.000001423324,0.000001593246,0.00009939531,0.0000255419,0.978129,0.0000365668,0.01719726,0.0001340748],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9889456,0.000394169,0.01010832,0.0000753538,0.00004052392,0.0003500949,0.0000164968,0.000005009326,0.0000644145],"genre_scores_gemma":[0.991711,0.00009879954,0.007649077,0.0001512791,0.00007788709,0.0001111109,0.00001293834,0.0000197286,0.0001681481],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.0170985,"threshold_uncertainty_score":0.3256016,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.01401902917178516,"score_gpt":0.3076732432123441,"score_spread":0.293654214040559,"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."}}