{"id":"W2006032038","doi":"10.1038/mt.2009.31","title":"Evidence for Long-term Efficacy and Safety of Gene Therapy for Wiskott–Aldrich Syndrome in Preclinical Models","year":2009,"lang":"en","type":"article","venue":"Molecular Therapy","topic":"Virus-based gene therapy research","field":"Biochemistry, Genetics and Molecular Biology","cited_by":84,"is_retracted":false,"has_abstract":false,"ca_institutions":"Mount Sinai Hospital","funders":"Fondazione Telethon","keywords":"Genetic enhancement; Wiskott–Aldrich syndrome; Wiskott–Aldrich syndrome protein; Immunology; Primary immunodeficiency; Haematopoiesis; Severe combined immunodeficiency; Transgene; Cancer research; Cell therapy; Biology; Autoimmunity; Bone marrow; Medicine; Stem cell; Gene; Cell; Immune system; Genetics; Actin cytoskeleton","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":["metaepi_narrow"],"consensus_categories":[],"category_scores_codex":[0.0008496154,0.0002752731,0.0003995423,0.0001051104,0.00006472365,0.00002658849,0.0004217635,0.0002543681,0.000007948974],"category_scores_gemma":[0.0001001598,0.0002551222,0.0002159709,0.0001615917,0.0001195691,0.00001296937,0.00003072732,0.0001185672,6.63731e-7],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00002115121,"about_ca_system_score_gemma":0.0001353853,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.000003724516,"about_ca_topic_score_gemma":0.00001112837,"domain_scores_codex":[0.9979855,0.0001774969,0.0004803912,0.0006697196,0.0002313791,0.0004554566],"domain_scores_gemma":[0.9987831,0.0001525444,0.0001240482,0.0006577601,0.0001675431,0.0001150193],"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":[0.02569187,0.0003923512,0.0007898878,0.00002295537,0.0001361133,0.000008467217,0.00006003695,0.000745683,0.8787838,0.00005358448,0.00001920388,0.09329604],"study_design_scores_gemma":[0.01144364,0.01988313,0.02172941,0.00009576872,0.00001373991,0.00002255705,0.000004160751,0.0006019943,0.9442936,0.001322741,0.0001962903,0.0003929871],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.936702,0.03174781,0.02854199,0.0005801388,0.00003688837,0.002336356,0.00002810527,0.00001257745,0.00001410332],"genre_scores_gemma":[0.9820902,0.01455798,0.002110799,0.0007746618,0.00005938575,0.0002067971,0.00008333741,0.00005380786,0.00006305935],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.09290306,"threshold_uncertainty_score":0.9999901,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.0900676855080766,"score_gpt":0.3958785833543971,"score_spread":0.3058108978463205,"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."}}