{"id":"W4407976740","doi":"10.1016/j.jtct.2025.01.039","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":2025,"lang":"en","type":"article","venue":"Transplantation and Cellular Therapy","topic":"Hemoglobinopathies and Related Disorders","field":"Medicine","cited_by":1,"is_retracted":false,"has_abstract":false,"ca_institutions":"Ottawa Hospital","funders":"","keywords":"Fetal hemoglobin; Hemoglobin; Medicine; Disease; Hemoglobin s; Cell; Normalization (sociology); Clinical trial; Internal medicine; Fetus; Sickle cell anemia; Chemistry; Pregnancy; Biology; Biochemistry; 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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0007439283,0.0002589125,0.0006949828,0.0001481615,0.000174878,0.0004783987,0.0003060832,0.0004631247,0.002070682],"category_scores_gemma":[0.001133327,0.00006169171,0.0004145453,0.0001519619,0.0002982091,0.0002233171,0.0001826395,0.001559027,0.0002367524],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0003733229,"about_ca_system_score_gemma":0.0003704846,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.000416401,"about_ca_topic_score_gemma":0.001143571,"domain_scores_codex":[0.9997205,0.0001399411,0.00001303617,0.00003143912,0.00004472139,0.00005043505],"domain_scores_gemma":[0.9997576,0.0001022594,0.00003242204,0.00002286413,0.00002176642,0.00006303755],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"design_other","study_design_gemma":"nonrandomized_trial","study_design_scores_codex":[0.1591405,0.003655228,0.007708383,0.001077068,0.00214661,0.002091523,0.0004580203,0.002867529,0.0874721,0.006295171,0.02347426,0.7036136],"study_design_scores_gemma":[0.2717254,0.1916077,0.06576374,0.0008561373,0.006234754,0.01099695,0.0005049043,0.01361364,0.1659771,0.009293801,0.2631729,0.0002529078],"study_design_candidate":"nonrandomized_trial","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9500445,0.02629196,0.003337976,0.004647995,0.0009047314,0.0002553161,0.000816291,0.0005415949,0.01315952],"genre_scores_gemma":[0.98974,0.003370217,0.001517003,0.001366793,0.0001084654,0.00007024681,0.0005257275,0.00003900788,0.003262601],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.002070682,"threshold_uncertainty_score":0.006927192,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.005268208090287432,"score_gpt":0.2161090230334272,"score_spread":0.2108408149431398,"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."}}