{"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":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0004112405,0.0001720703,0.0001858935,0.00002401395,0.0005354501,0.00003237099,0.0003885399,0.00004518918,0.00006342305],"category_scores_gemma":[0.00007307268,0.000123588,0.0003334847,0.0001033924,0.00003302384,0.000001826737,0.0002257689,0.00008226722,0.000004424387],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00004808594,"about_ca_system_score_gemma":0.0002281779,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0001508183,"about_ca_topic_score_gemma":0.00009448457,"domain_scores_codex":[0.9986073,0.0001204329,0.000236134,0.000429396,0.0002714912,0.0003352934],"domain_scores_gemma":[0.9989569,0.00007300733,0.00008475455,0.0008047155,0.00003691547,0.00004375676],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"bench_or_experimental","study_design_gemma":"bench_or_experimental","study_design_scores_codex":[0.002311683,0.003387761,0.0711419,0.000007484274,0.001865184,0.0001342538,0.0006794091,0.0007851198,0.9127564,0.00004525255,0.002316231,0.004569251],"study_design_scores_gemma":[0.0344642,0.02975299,0.0241766,0.00001754972,0.0008424451,0.0002016246,0.00270284,0.0002248199,0.7146478,0.0007406182,0.1907793,0.001449212],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9950662,0.002709119,0.00006366932,0.0006774451,0.0002266357,0.0007159748,0.0002112955,0.00002113993,0.0003085507],"genre_scores_gemma":[0.9964607,0.00002474068,0.0001469605,0.0003552648,0.00005504702,0.0006365039,0.0005666887,0.00003457658,0.001719547],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.1981087,"threshold_uncertainty_score":0.5039773,"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."}}