{"id":"W2291715119","doi":"10.1089/hum.2015.137","title":"Gene Therapy for X-Linked Severe Combined Immunodeficiency: Where Do We Stand?","year":2016,"lang":"en","type":"review","venue":"Human Gene Therapy","topic":"Virus-based gene therapy research","field":"Biochemistry, Genetics and Molecular Biology","cited_by":108,"is_retracted":false,"has_abstract":true,"ca_institutions":"Pyrogenesis (Canada)","funders":"Agence Nationale de la Recherche; European Commission; French Muscular Dystrophy Association; Muscular Dystrophy Association","keywords":"Genetic enhancement; Severe combined immunodeficiency; Retrovirus; Vector (molecular biology); Viral vector; Lentivirus; Vectors in gene therapy; Immunodeficiency; Hematopoietic stem cell; Immunology; Immune system; Gene delivery; Medicine; Adverse effect; Biology; Haematopoiesis; Gene; Virology; Human immunodeficiency virus (HIV); Stem cell; Genetics; Virus; Internal medicine; Viral disease","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":["metaepi_narrow"],"category_scores_codex":[0.001102035,0.00134525,0.001994758,0.0003015399,0.0006130199,0.0001941216,0.00202026,0.001106049,0.0007958029],"category_scores_gemma":[0.00001839509,0.0009835743,0.001544422,0.000312754,0.0003270351,0.00001175386,0.0002131112,0.0004529642,0.0001292675],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0001818186,"about_ca_system_score_gemma":0.001202138,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.000005714054,"about_ca_topic_score_gemma":0.0000153815,"domain_scores_codex":[0.9946317,0.0006760383,0.001131636,0.001690352,0.0005523704,0.001317915],"domain_scores_gemma":[0.9960174,0.0001023414,0.0006369397,0.002584677,0.0004052428,0.0002533788],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"design_other","study_design_gemma":"not_applicable","study_design_scores_codex":[0.002783249,0.0003221794,0.000001367641,0.0009769842,0.001153235,0.00000663512,0.00007259384,0.000001749428,0.1244666,0.00004196832,0.003914329,0.8662591],"study_design_scores_gemma":[0.005622155,0.008866602,0.000001474898,0.001462374,0.00006796607,0.00003974604,0.00001164757,7.195835e-7,0.02144423,0.0004693633,0.9607059,0.001307763],"study_design_candidate":"not_applicable","study_design_consensus":null,"genre_codex":"review","genre_gemma":"review","genre_scores_codex":[0.00653143,0.9875094,0.0001572642,0.0001200192,0.0003705216,0.004253628,0.0007417393,0.00009604537,0.0002199885],"genre_scores_gemma":[0.0002675297,0.9893935,0.0002079331,0.0001201547,0.001247412,0.001417675,0.0007560399,0.0005290053,0.006060733],"genre_candidate":"review","genre_consensus":"review","teacher_disagreement_score":0.9567916,"threshold_uncertainty_score":0.9999298,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.06359927609771422,"score_gpt":0.3682589439071943,"score_spread":0.3046596678094801,"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."}}