{"id":"W4281750080","doi":"10.1182/blood.2022016196","title":"Outcomes following treatment for ADA-deficient severe combined immunodeficiency: a report from the PIDTC","year":2022,"lang":"en","type":"article","venue":"Blood","topic":"Virus-based gene therapy research","field":"Biochemistry, Genetics and Molecular Biology","cited_by":50,"is_retracted":false,"has_abstract":true,"ca_institutions":"Université de Montréal; Centre Hospitalier Universitaire Sainte-Justine; University of Manitoba; BC Children's Hospital; CancerCare Manitoba","funders":"NHLBI Division of Intramural Research; National Center for Advancing Translational Sciences; National Institute of Neurological Disorders and Stroke; National Institute of Allergy and Infectious Diseases; Office of Naval Research; Health Resources and Services Administration; National Cancer Institute; National Institutes of Health; Rare Diseases Clinical Research Network; National Heart, Lung, and Blood Institute; St. Baldrick's Foundation","keywords":"Medicine; Enzyme replacement therapy; Internal medicine; Adenosine deaminase deficiency; Hematopoietic cell; Hematopoietic stem cell transplantation; Severe combined immunodeficiency; Gastroenterology; Adenosine deaminase; Transplantation; Disease; Haematopoiesis; Stem cell; Biology; Adenosine","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":[0.0004512267,0.0001139486,0.000194273,0.0002620751,0.0002813114,0.0004036291,0.0001210568,0.0001822341,0.000354974],"category_scores_gemma":[0.002132272,0.00005048617,0.0002230035,0.0004884874,0.0001770268,0.0001534861,0.0002027175,0.0003252516,0.0000542323],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0004668859,"about_ca_system_score_gemma":0.0003536331,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.003099802,"about_ca_topic_score_gemma":0.002875947,"domain_scores_codex":[0.9996954,0.00008881253,0.00003852132,0.00003836163,0.00008659499,0.00005230658],"domain_scores_gemma":[0.9991826,0.0002440984,0.0003537521,0.00004180302,0.00008936989,0.00008838347],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"observational","study_design_gemma":"observational","study_design_scores_codex":[0.0003687601,0.00007390057,0.9606904,0.00009041544,0.00007854389,0.003225697,0.0003860228,0.0001375461,0.0004585098,0.0001064682,0.001217466,0.03316628],"study_design_scores_gemma":[0.00004019176,0.0004383855,0.9867143,0.00009551566,0.0001298655,0.008761964,0.0002787366,0.0001912731,0.0003651353,0.0000678787,0.002906954,0.000009715096],"study_design_candidate":"observational","study_design_consensus":"observational","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9885148,0.007500533,0.0002768992,0.0003588603,0.00002338252,0.00002026733,0.0003296649,0.00001097886,0.002964624],"genre_scores_gemma":[0.9977909,0.001538677,0.00006615693,0.000126219,0.00003270792,0.000007258559,0.000309749,0.000001689244,0.0001267336],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.003099802,"threshold_uncertainty_score":0.006163538,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.02227283479849256,"score_gpt":0.295437949642415,"score_spread":0.2731651148439225,"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."}}