{"id":"W2562772129","doi":"10.1016/s1525-0016(16)33908-9","title":"299. Efficient Functional Oxidase Correction after CRISPR-Oligomer Gene Repair in X-CGD Patient Hematopoietic Stem Cells (HSC) Using Non-Viral, cGMP Compliant, Scalable, Closed System","year":2015,"lang":"en","type":"article","venue":"Molecular Therapy","topic":"CRISPR and Genetic Engineering","field":"Biochemistry, Genetics and Molecular Biology","cited_by":0,"is_retracted":false,"has_abstract":true,"ca_institutions":"","funders":"","keywords":"Biology; Genome editing; CRISPR; Genetic enhancement; Stem cell; Hematopoietic stem cell; Viral vector; Cas9; Gene; Haematopoiesis; Molecular biology; Genetics","routes":{"ca_aff":false,"ca_fund":false,"ca_venue":false,"about_ca":true,"invisible_to_affiliation_only":true},"retraction":null,"screen":null,"direct_labels":[],"prediction":{"model_version":"codex-gemma-dda1882f352a","candidate_categories":["metaepi_narrow"],"consensus_categories":[],"category_scores_codex":[0.0002445455,0.0003452766,0.0002964397,0.0001412969,0.00006575521,0.00003446565,0.0001294246,0.0002078863,0.00001025049],"category_scores_gemma":[0.000005437156,0.0003437102,0.0002104498,0.000227846,0.00004905928,0.000005511674,0.00008201441,0.0001516721,0.00002830715],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0001440031,"about_ca_system_score_gemma":0.00009607607,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00008257228,"about_ca_topic_score_gemma":0.000005600773,"domain_scores_codex":[0.9980397,0.0001236906,0.0004465929,0.0006022573,0.0003482297,0.0004394889],"domain_scores_gemma":[0.9989865,0.000007931701,0.0001013929,0.0005573355,0.0001522549,0.0001945916],"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":[0.0003492792,0.0001847457,0.0003927526,0.00004488975,0.00006834046,0.00008514814,0.0001879231,0.1305971,0.8670104,0.000003404909,0.0004974437,0.0005786107],"study_design_scores_gemma":[0.001596329,0.0003397583,0.0005950454,0.00008508044,0.00002363285,0.00009979265,0.0003049884,0.04888381,0.9453106,0.000002143928,0.002333946,0.0004249022],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.8814715,0.003404136,0.1133918,0.00001511605,0.001084043,0.0004978746,0.00001840511,0.00004680178,0.00007037164],"genre_scores_gemma":[0.9983631,0.0000405459,0.0009424656,0.0002454546,0.0001079725,0.00008756527,0.00004188159,0.00007278312,0.00009824374],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.1168916,"threshold_uncertainty_score":0.9999015,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.01357935490362445,"score_gpt":0.2534261298596158,"score_spread":0.2398467749559914,"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."}}