{"id":"W3097957926","doi":"10.1182/blood-2020-139575","title":"Safety and Efficacy of CTX001 in Patients with Transfusion-Dependent β-Thalassemia and Sickle Cell Disease: Early Results from the Climb THAL-111 and Climb SCD-121 Studies of Autologous CRISPR-CAS9-Modified CD34+ Hematopoietic Stem and Progenitor Cells","year":2020,"lang":"en","type":"article","venue":"Blood","topic":"Hemoglobinopathies and Related Disorders","field":"Medicine","cited_by":51,"is_retracted":false,"has_abstract":true,"ca_institutions":"Hospital for Sick Children; BC Children's Hospital","funders":"","keywords":"Climb; Thalassemia; Medicine; CRISPR; Disease; Sickle cell anemia; Blood transfusion; Internal medicine; Biology; Genetics; Gene","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":[0.0001745923,0.0002485375,0.000620836,0.00004492631,0.00009697971,0.00001580844,0.00007028817,0.0001327155,0.000001859668],"category_scores_gemma":[0.00007015274,0.0001537696,0.00004244239,0.0001523796,0.0003501553,0.00005169951,0.000104129,0.0002489923,4.203957e-7],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000008977006,"about_ca_system_score_gemma":0.00006010089,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0001114496,"about_ca_topic_score_gemma":0.00002264755,"domain_scores_codex":[0.9983018,0.0001169404,0.0005552237,0.0004771773,0.0003128135,0.0002360301],"domain_scores_gemma":[0.998844,0.0003990276,0.000190546,0.0002395271,0.0000912232,0.0002356493],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"observational","study_design_gemma":"observational","study_design_scores_codex":[0.01600481,0.002690251,0.938403,0.001830222,0.0006838269,0.0001738692,0.03233522,0.00001669943,0.004812784,0.00001818788,0.00005406883,0.002977093],"study_design_scores_gemma":[0.04526289,0.002903603,0.9335447,0.0008887635,0.001520165,0.00001086268,0.007331848,0.00008130084,0.008111464,0.0000213975,0.00002784094,0.0002951377],"study_design_candidate":"observational","study_design_consensus":"observational","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9878896,0.009770162,0.000003366199,0.0007215677,0.00003543683,0.001258013,0.0002291832,0.00002150847,0.000071105],"genre_scores_gemma":[0.9958021,0.003979358,0.00006526988,0.00006364064,0.00002026129,0.000009676046,0.00001661275,0.00002489981,0.00001818078],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.02925809,"threshold_uncertainty_score":0.6270543,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.01139060119781103,"score_gpt":0.2249172256761326,"score_spread":0.2135266244783216,"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."}}