{"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":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0001494687,0.0001372178,0.000226548,0.00004299701,0.00002244799,0.000007272139,0.0001610903,0.0000760867,0.000009402162],"category_scores_gemma":[0.00002715748,0.0001448153,0.0001221143,0.0000649121,0.00005411519,0.000002492966,0.00005972301,0.0001140522,1.141156e-7],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000007796548,"about_ca_system_score_gemma":0.0001136209,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.000006279231,"about_ca_topic_score_gemma":0.000008342346,"domain_scores_codex":[0.9988728,0.00006655583,0.0004009885,0.0003242597,0.0001471057,0.0001883467],"domain_scores_gemma":[0.9994029,0.00001135499,0.0001055867,0.000338796,0.00009364383,0.0000476666],"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":[0.00003715329,0.0002525238,0.004073261,0.00001963975,0.00002165126,0.000002705179,0.0001020111,0.04300546,0.9521045,0.00003747374,0.00001131105,0.0003323273],"study_design_scores_gemma":[0.00138737,0.0003758637,0.003579203,0.00001227354,0.000006634652,0.00002062288,0.0000624728,0.01723453,0.9771616,0.00003348305,0.00001485847,0.0001110633],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.911656,0.0003807863,0.0875221,0.000006747226,0.00005171817,0.0001293022,0.00002083703,0.000005976843,0.000226519],"genre_scores_gemma":[0.9132552,0.0001094072,0.08620894,0.00002493141,0.0002083781,0.00002137041,0.00007035005,0.00002186644,0.00007952035],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.02577092,"threshold_uncertainty_score":0.5905395,"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."}}