{"id":"W4377136750","doi":"10.1007/s40123-023-00729-6","title":"ABE8e Corrects Pax6-Aniridic Variant in Humanized Mouse ESCs and via LNPs in Ex Vivo Cortical Neurons","year":2023,"lang":"en","type":"article","venue":"Ophthalmology and Therapy","topic":"Retinal Development and Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":7,"is_retracted":false,"has_abstract":true,"ca_institutions":"Incisive Genetics (Canada); BC Children's Hospital; University of British Columbia","funders":"British Columbia Knowledge Development Fund; University of British Columbia; BC Children's Hospital; Canadian Institutes of Health Research; BC Children’s Hospital Foundation","keywords":"Ex vivo; In vivo; Humanized mouse; Biology; Molecular biology; Neuroscience; Genetics","routes":{"ca_aff":true,"ca_fund":true,"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":[0.0002137351,0.0001464539,0.0001837483,0.00009390432,0.00006154292,0.00001323037,0.00008232016,0.0001828595,0.00006368551],"category_scores_gemma":[0.00004007382,0.000135649,0.00002464895,0.0001469457,0.0001340724,0.000004513836,0.00006653227,0.0001365396,0.000009561799],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000004065736,"about_ca_system_score_gemma":0.00003219873,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00005381788,"about_ca_topic_score_gemma":0.00002426607,"domain_scores_codex":[0.9990181,0.0001329014,0.0001786459,0.0003421147,0.00005319229,0.0002749888],"domain_scores_gemma":[0.9997226,0.00004519204,0.00003225301,0.0001304758,0.00001314696,0.00005630827],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"bench_or_experimental","study_design_gemma":"observational","study_design_scores_codex":[0.0015238,0.0002903599,0.4425772,0.00002605226,0.00006332325,0.00184783,0.0005181066,0.00001245424,0.5475243,0.0004641744,0.0006181786,0.004534316],"study_design_scores_gemma":[0.005942519,0.001295384,0.9620555,0.00001998689,0.00000910031,0.001745203,0.0002341645,0.000411523,0.02170678,0.002896085,0.003106047,0.0005777002],"study_design_candidate":"observational","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9975626,0.0008343805,0.00001614804,0.0001882395,0.0001176814,0.0001774958,0.00000256862,0.00001136059,0.001089534],"genre_scores_gemma":[0.9963115,0.002668527,0.00004137478,0.0001937363,0.00002459365,0.00002342001,0.00003863413,0.00001665339,0.0006814864],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.5258175,"threshold_uncertainty_score":0.5531604,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.01599918362080245,"score_gpt":0.2670283437906876,"score_spread":0.2510291601698851,"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."}}