{"id":"W4407679695","doi":"10.1089/hum.2024.170","title":"Minimally Humanized <i>Ezh2</i> Exon-18 Mouse Cell Lines Validate Preclinical CRISPR/Cas9 Approach to Treat Weaver Syndrome","year":2025,"lang":"en","type":"article","venue":"Human Gene Therapy","topic":"CRISPR and Genetic Engineering","field":"Biochemistry, Genetics and Molecular Biology","cited_by":1,"is_retracted":false,"has_abstract":true,"ca_institutions":"BC Children's Hospital; University of British Columbia","funders":"","keywords":"CRISPR; Humanized mouse; Exon; Cas9; Gene knockin; Genome editing; Biology; Cancer research; Medicine; Genetics; 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":"codex-gemma-dda1882f352a","candidate_categories":["metaepi_narrow"],"consensus_categories":[],"category_scores_codex":[0.0002430222,0.00038837,0.0003938389,0.000120421,0.0001657235,0.00007603945,0.0004951628,0.0002492476,0.00008527756],"category_scores_gemma":[0.00001690264,0.0003707921,0.0002343272,0.0001586138,0.00006207413,0.000004693616,0.0001712096,0.0001513126,0.00003279887],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00001970709,"about_ca_system_score_gemma":0.00005628887,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00001737797,"about_ca_topic_score_gemma":0.000007612969,"domain_scores_codex":[0.9980714,0.00008572486,0.000459425,0.0007642227,0.0001682558,0.0004509417],"domain_scores_gemma":[0.9987962,0.00001948387,0.00006604648,0.0008663506,0.00009410912,0.0001577577],"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":[0.0002140533,0.0003576388,0.0002165439,0.00004914921,0.00009149463,0.000007415413,0.0001279363,0.0005385014,0.9881379,0.00002330872,0.009467209,0.0007688345],"study_design_scores_gemma":[0.002297722,0.000650096,0.00117392,0.00001374536,0.00004035716,0.00001795064,0.00004520182,0.00005339229,0.9665137,0.00004217412,0.02870662,0.0004451593],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9812298,0.002651698,0.008731747,0.00007210788,0.0001807469,0.0005732678,0.00002741492,0.00007682332,0.006456419],"genre_scores_gemma":[0.9713146,0.0007516653,0.004623178,0.00098673,0.0003197125,0.0001308809,0.0002094113,0.00008206206,0.02158176],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.02162425,"threshold_uncertainty_score":0.9998744,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.03130332561219439,"score_gpt":0.3465706260901964,"score_spread":0.315267300478002,"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."}}