{"id":"W4405038074","doi":"10.1182/blood-2024-210343","title":"Reni-Cel, an Investigational AsCas12a Gene-Edited Cell Medicine, Led to Sustained Hemoglobin Normalization and Increased Fetal Hemoglobin in Patients with Severe Sickle Cell Disease Treated in the RUBY Trial","year":2024,"lang":"en","type":"article","venue":"Blood","topic":"Prenatal Screening and Diagnostics","field":"Medicine","cited_by":7,"is_retracted":false,"has_abstract":true,"ca_institutions":"Ottawa Hospital","funders":"","keywords":"Fetal hemoglobin; Medicine; CD34; Stem cell; Genetic enhancement; Progenitor cell; Tolerability; Immunology; Cancer research; Internal medicine; Biology; Adverse effect; Gene; Fetus; Genetics","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":[],"consensus_categories":[],"category_scores_codex":[0.0003472178,0.0002668701,0.0003110015,0.0003021729,0.00006786025,0.00005897332,0.0001300214,0.0001094197,0.00002845204],"category_scores_gemma":[0.0005492468,0.0001802873,0.00003411682,0.0009553696,0.00009289017,0.0001925507,0.00004697231,0.0002214261,0.00000604854],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00007376226,"about_ca_system_score_gemma":0.0002541559,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0009392189,"about_ca_topic_score_gemma":0.0002127542,"domain_scores_codex":[0.9980683,0.000221032,0.0003982981,0.0004474856,0.0005529148,0.0003119316],"domain_scores_gemma":[0.9988372,0.0002605665,0.00006772881,0.0002940678,0.0001146055,0.0004258195],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"observational","study_design_gemma":"observational","study_design_scores_codex":[0.04001148,0.005271848,0.9451677,0.0005383518,0.00007753277,0.001193686,0.002393504,0.0003815721,0.001069254,0.0001699009,0.003123226,0.0006019273],"study_design_scores_gemma":[0.1591953,0.00412242,0.8274545,0.0006590199,0.0006656332,0.00002645518,0.0004633875,0.004346532,0.0023243,0.0001204963,0.0001460238,0.0004759176],"study_design_candidate":"observational","study_design_consensus":"observational","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.996557,0.0002376114,0.00004496169,0.0006840148,0.00006710247,0.001576736,0.0001440685,0.0000909106,0.0005976257],"genre_scores_gemma":[0.9958657,0.000007767208,0.0003741389,0.0005157095,0.000254119,0.00008547547,0.002769576,0.00003307208,0.00009444946],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.1191838,"threshold_uncertainty_score":0.7351901,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.006859896190244973,"score_gpt":0.2220551658836929,"score_spread":0.215195269693448,"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."}}