{"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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0001874643,0.0002347534,0.0002235924,0.0001936049,0.0001909897,0.000313979,0.0002416439,0.0003661776,0.002447629],"category_scores_gemma":[0.0001645671,0.0001227753,0.0002143416,0.0001265821,0.0001946741,0.0001375507,0.0002011171,0.0003459779,0.001406985],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0002494846,"about_ca_system_score_gemma":0.0003131527,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0006673266,"about_ca_topic_score_gemma":0.0007208968,"domain_scores_codex":[0.9997877,0.00002664208,0.00002043436,0.00004906396,0.00009744458,0.000018699],"domain_scores_gemma":[0.9999263,0.00001178401,0.00002177965,0.00001208699,0.00001363593,0.00001443384],"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":[0.00004544637,0.00001879932,0.0001421191,0.00002694134,0.000001959625,0.0001009052,0.00001369926,0.00009129962,0.9961213,0.0002434171,0.0002930081,0.00290094],"study_design_scores_gemma":[0.00001110078,0.0000953597,0.001017404,0.000003387979,0.000003607806,0.0004136402,0.000005735064,0.0009390104,0.9922691,0.00004390893,0.005191834,0.000005889888],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.8729883,0.001072772,0.1018522,0.0003584012,0.0002300496,0.0004624409,0.00291198,0.00310488,0.01701884],"genre_scores_gemma":[0.9502221,0.0004845228,0.03153913,0.0001301387,0.0000143779,0.0001864818,0.002285741,0.000153165,0.01498433],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.002447629,"threshold_uncertainty_score":0.008188128,"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."}}