{"id":"W4417006170","doi":"10.1182/blood-2025-4314","title":"CRISPR-Cas12a gene editing of the HBG1/2 promoters leads to sustained normalization of total hemoglobin and increased fetal hemoglobin in patients with severe sickle cell disease: Updated Results from the RUBY trial","year":2025,"lang":"en","type":"article","venue":"Blood","topic":"Hemoglobinopathies and Related Disorders","field":"Medicine","cited_by":1,"is_retracted":false,"has_abstract":true,"ca_institutions":"Ottawa Hospital","funders":"","keywords":"Fetal hemoglobin; Stem cell; Haematopoiesis; Progenitor cell; Hematopoietic stem cell transplantation; Sickle cell anemia; Anemia; Hemoglobin","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":[0.002272181,0.0004801487,0.001192641,0.0002247926,0.0001832612,0.0009705094,0.0003613356,0.0005647089,0.003501453],"category_scores_gemma":[0.001867861,0.000113684,0.001178188,0.0003146812,0.0003118737,0.0004894823,0.0002788228,0.001788029,0.0005019174],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0004656097,"about_ca_system_score_gemma":0.0005072798,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0005331722,"about_ca_topic_score_gemma":0.001345629,"domain_scores_codex":[0.9993733,0.0002885677,0.00006543531,0.00007565133,0.000121719,0.00007537363],"domain_scores_gemma":[0.9990928,0.0003566095,0.0001996596,0.00005462997,0.000160849,0.0001354197],"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":[0.1173796,0.002497959,0.006698071,0.007310252,0.005369307,0.0003914079,0.0003023144,0.002841606,0.02727513,0.001937199,0.07410566,0.7538915],"study_design_scores_gemma":[0.3858135,0.1431509,0.05611312,0.005399554,0.01809353,0.003807938,0.0003743676,0.004587191,0.02480435,0.007781523,0.3497868,0.0002872896],"study_design_candidate":"nonrandomized_trial","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.6495345,0.2757431,0.007706052,0.01565954,0.002807878,0.002338179,0.01444046,0.000675094,0.03109519],"genre_scores_gemma":[0.8536861,0.09693135,0.007239171,0.01317256,0.001402263,0.002711853,0.01447673,0.0002362905,0.01014367],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.003501453,"threshold_uncertainty_score":0.01201665,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.002875867535686232,"score_gpt":0.1958532900947138,"score_spread":0.1929774225590276,"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."}}