{"id":"W3197279604","doi":"10.1089/hum.2021.152","title":"Preclinical Assessment of a Gene-Editing Approach in a Mouse Model of Mitochondrial Neurogastrointestinal Encephalomyopathy","year":2021,"lang":"en","type":"article","venue":"Human Gene Therapy","topic":"CRISPR and Genetic Engineering","field":"Biochemistry, Genetics and Molecular Biology","cited_by":16,"is_retracted":false,"has_abstract":true,"ca_institutions":"Acuitas Therapeutics (Canada)","funders":"","keywords":"Thymidine phosphorylase; Biology; CRISPR; Transgene; Molecular biology; Cas9; Gene; Genetic enhancement; Genome editing; Genetics; Biochemistry; Enzyme","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":[0.0007988418,0.001265863,0.0008449873,0.0008838493,0.0003586906,0.0006989411,0.0007532,0.001425001,0.001788887],"category_scores_gemma":[0.0003247373,0.0003032505,0.0007142206,0.0002529132,0.0005555398,0.0006854963,0.0003149138,0.002960058,0.0006144646],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0004264426,"about_ca_system_score_gemma":0.0005357338,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0006030621,"about_ca_topic_score_gemma":0.001232711,"domain_scores_codex":[0.9996132,0.00006854998,0.00003279842,0.0001007203,0.00009445598,0.0000902531],"domain_scores_gemma":[0.9995466,0.00005281112,0.00009540412,0.00005342314,0.00007866784,0.0001730576],"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":[0.001479371,0.002919397,0.0002379439,0.0002197306,0.00004835839,0.0002164751,0.00007162416,0.0005002309,0.9885089,0.000385225,0.0003924035,0.005020359],"study_design_scores_gemma":[0.0006965533,0.04962989,0.002138045,0.00009086219,0.0002670524,0.0006485104,0.0001086948,0.002196778,0.9318742,0.0003030751,0.01200776,0.00003858507],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9721661,0.003680312,0.01548993,0.0005431826,0.0004477899,0.001184396,0.001767768,0.0007115093,0.004009051],"genre_scores_gemma":[0.9629843,0.005043258,0.01352321,0.0004110195,0.00009723378,0.002504967,0.003459259,0.0001107034,0.01186608],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.001788887,"threshold_uncertainty_score":0.005984426,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.03661134595965766,"score_gpt":0.3505459935657665,"score_spread":0.3139346476061089,"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."}}