{"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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0008463302,0.0002277821,0.0004830217,0.0001357637,0.000170833,0.0003942906,0.0001611254,0.0003122396,0.001548746],"category_scores_gemma":[0.0007296556,0.00006633923,0.0004922278,0.0001422344,0.0002908322,0.0002196966,0.0001846204,0.000779648,0.0002169818],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0003664205,"about_ca_system_score_gemma":0.0005205984,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0002598931,"about_ca_topic_score_gemma":0.0004916204,"domain_scores_codex":[0.9997669,0.00008364571,0.0000249859,0.00003633527,0.00006478111,0.00002331524],"domain_scores_gemma":[0.9994111,0.0001542621,0.0001926336,0.00003333716,0.00007527047,0.0001333089],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"design_other","study_design_gemma":"observational","study_design_scores_codex":[0.1182369,0.01000874,0.123021,0.002395777,0.002017125,0.001544237,0.0006881208,0.003232395,0.1403237,0.0008778097,0.01102898,0.586625],"study_design_scores_gemma":[0.03522744,0.5539776,0.259112,0.0006365904,0.003129923,0.01052535,0.0003735365,0.003933481,0.09185246,0.0006336403,0.04048462,0.0001133446],"study_design_candidate":"observational","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9881684,0.0076746,0.0008597768,0.0003136399,0.00003956167,0.0002142785,0.0006512898,0.00003997571,0.002038344],"genre_scores_gemma":[0.9944373,0.002289537,0.0007865921,0.0004132847,0.00004359148,0.0001534116,0.001188238,0.00000742896,0.0006804412],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.001548746,"threshold_uncertainty_score":0.005181015,"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."}}