{"id":"W2783766595","doi":"10.1016/j.jaci.2017.11.050","title":"T-cell gene therapy for perforin deficiency corrects cytotoxicity defects and prevents hemophagocytic lymphohistiocytosis manifestations","year":2018,"lang":"en","type":"article","venue":"Journal of Allergy and Clinical Immunology","topic":"Autoimmune and Inflammatory Disorders Research","field":"Medicine","cited_by":57,"is_retracted":false,"has_abstract":true,"ca_institutions":"Institute of Infection and Immunity","funders":"Medical Research Council; National Institute for Health and Care Research; Action Medical Research; Wellcome Trust","keywords":"Perforin; Cytotoxic T cell; Lymphocytic choriomeningitis; CD8; Hemophagocytic lymphohistiocytosis; Genetic enhancement; Cancer research; Biology; Cytotoxicity; Immunology; Virology; Molecular biology; Immune system; Medicine; Gene; In vitro; Disease; Pathology; 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":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0006946171,0.0001291773,0.0004548324,0.0001172217,0.0002190742,0.0000162211,0.0001147762,0.0002026701,0.00007904191],"category_scores_gemma":[0.0004264338,0.0001002592,0.0001635907,0.00008454781,0.0005073851,0.0001004141,0.00005452711,0.0002917225,0.000007681849],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00003942395,"about_ca_system_score_gemma":0.0003701458,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.000008184576,"about_ca_topic_score_gemma":0.000006493825,"domain_scores_codex":[0.9985304,0.0001390359,0.000747152,0.0001884511,0.0001403607,0.0002545972],"domain_scores_gemma":[0.9986275,0.0004168666,0.0003418477,0.0001577873,0.0003384123,0.0001175209],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"bench_or_experimental","study_design_gemma":"observational","study_design_scores_codex":[0.0515955,0.01351632,0.2493391,0.0005453421,0.007455327,0.0007051937,0.003967306,0.00002000041,0.3525708,0.002036703,0.008391699,0.3098567],"study_design_scores_gemma":[0.0321455,0.03955341,0.8749545,0.0002049543,0.0003053517,0.002392695,0.001028163,0.0009167324,0.03444022,0.00141246,0.0122187,0.0004273309],"study_design_candidate":"observational","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9897719,0.008419962,0.000605363,0.0002361553,0.0003141465,0.0002614429,0.00000205733,0.000008111372,0.0003808711],"genre_scores_gemma":[0.976436,0.02178734,0.0007592366,0.0003145531,0.000161364,0.000008188729,0.000003064671,0.00001505199,0.0005152427],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.6256154,"threshold_uncertainty_score":0.4088452,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.03584597955059322,"score_gpt":0.3485100994651467,"score_spread":0.3126641199145535,"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."}}