{"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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.004210741,0.0005257231,0.0007829753,0.0007796112,0.000321618,0.0006488251,0.0008368531,0.001540868,0.003441638],"category_scores_gemma":[0.003510699,0.0002244027,0.0007600033,0.0003354507,0.001218781,0.001261406,0.0003049312,0.001386001,0.000810852],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0004657337,"about_ca_system_score_gemma":0.0005794206,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0004055371,"about_ca_topic_score_gemma":0.0004849297,"domain_scores_codex":[0.9992036,0.0002487994,0.0001025757,0.0001214417,0.0002332119,0.00009035009],"domain_scores_gemma":[0.9929593,0.003567315,0.001090841,0.001089041,0.0009253789,0.0003680557],"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":[0.02253148,0.005365824,0.0113439,0.002684486,0.00127632,0.0009579971,0.0002529891,0.001570389,0.7787427,0.004921173,0.004256594,0.1660961],"study_design_scores_gemma":[0.00409019,0.08830432,0.05873934,0.001058363,0.002335345,0.004624547,0.0002822549,0.002596674,0.7864525,0.006945344,0.04444812,0.0001230108],"study_design_candidate":"observational","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.8626731,0.1049492,0.01433793,0.005071371,0.0006896968,0.0002870642,0.001844487,0.0004049601,0.009742224],"genre_scores_gemma":[0.9656442,0.02350339,0.002894963,0.0009401549,0.000331365,0.0002934704,0.002026188,0.00007426507,0.004292052],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.004210741,"threshold_uncertainty_score":0.02226877,"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."}}