{"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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0008219281,0.0005869005,0.0005039491,0.0004755689,0.0003111235,0.0007221771,0.0006388349,0.0009961288,0.004652022],"category_scores_gemma":[0.0005884425,0.000282008,0.0005201368,0.0002372766,0.0004923186,0.0003964767,0.0003749493,0.002685102,0.002146722],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0004246281,"about_ca_system_score_gemma":0.0004437763,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.001160627,"about_ca_topic_score_gemma":0.001870299,"domain_scores_codex":[0.9989223,0.0001436603,0.0001369371,0.0002394487,0.0004362909,0.0001213045],"domain_scores_gemma":[0.9995245,0.00009557067,0.0001074433,0.00009931855,0.00008318359,0.00009001978],"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":[0.0001399288,0.0001732469,0.0003093143,0.00008625631,0.00002993373,0.0002129007,0.0000676656,0.0003612924,0.9932162,0.001144653,0.0009719905,0.003286591],"study_design_scores_gemma":[0.0000623049,0.0005970038,0.00108859,0.00002340357,0.00004754209,0.0006265512,0.00004330899,0.001279347,0.9772668,0.0002428479,0.01870763,0.00001473227],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.8342628,0.003865522,0.1279771,0.00152763,0.0009099211,0.001063057,0.01246024,0.002710109,0.01522365],"genre_scores_gemma":[0.9160171,0.002887877,0.04783209,0.000826298,0.00006476203,0.001390678,0.009993575,0.000748543,0.02023908],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.004652022,"threshold_uncertainty_score":0.01556259,"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."}}