{"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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.001799653,0.0005917487,0.001073297,0.0001555463,0.0002715155,0.0008275365,0.0004928857,0.001033197,0.006291524],"category_scores_gemma":[0.001688644,0.0001406884,0.001178137,0.0002011395,0.000380936,0.0005510548,0.0002542485,0.002915725,0.0007591242],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0004498497,"about_ca_system_score_gemma":0.0007192132,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0005596512,"about_ca_topic_score_gemma":0.001635396,"domain_scores_codex":[0.9994554,0.0002760953,0.00002912289,0.00007127188,0.00007229794,0.00009582141],"domain_scores_gemma":[0.999343,0.000206952,0.0001171997,0.00005319996,0.00006621228,0.0002133902],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"randomized_trial","study_design_gemma":"nonrandomized_trial","study_design_scores_codex":[0.4502285,0.009163328,0.005095951,0.002899686,0.003641475,0.001220399,0.0005683212,0.004959861,0.04210902,0.003757347,0.06011266,0.4162433],"study_design_scores_gemma":[0.6097324,0.2441616,0.02287051,0.000731332,0.003199396,0.003022559,0.0001790208,0.006228291,0.0207563,0.003485524,0.08551776,0.0001152907],"study_design_candidate":"nonrandomized_trial","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.906335,0.03225202,0.005175786,0.007645335,0.001517069,0.002522246,0.008848362,0.001133024,0.03457119],"genre_scores_gemma":[0.9697263,0.00624827,0.002912535,0.004177518,0.000368022,0.001083963,0.005475192,0.0001014148,0.009906755],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.006291524,"threshold_uncertainty_score":0.02104729,"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."}}