{"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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0005243389,0.0005476609,0.0009334201,0.001229756,0.000214696,0.0007910809,0.0006608205,0.0008712117,0.001973045],"category_scores_gemma":[0.0005352196,0.000154854,0.0003015797,0.001299727,0.0005475479,0.001320226,0.0004093694,0.001680227,0.001291892],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0003801351,"about_ca_system_score_gemma":0.0007098628,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0006181732,"about_ca_topic_score_gemma":0.001199711,"domain_scores_codex":[0.9998957,0.00002127378,0.00001448572,0.00001640465,0.0000410653,0.00001092956],"domain_scores_gemma":[0.9997815,0.000113401,0.00002760404,0.000007441973,0.00004809453,0.00002177002],"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":[0.00005761945,0.00004868801,0.0001169489,0.00865038,0.0000451566,0.0001399651,0.00007613133,0.0001882324,0.001880134,0.003680132,0.02017774,0.9649389],"study_design_scores_gemma":[0.00001665183,0.0001080243,0.0005080381,0.002865975,0.00006481749,0.001183732,0.00008165687,0.00005685881,0.0006148191,0.002588113,0.9918966,0.00001462916],"study_design_candidate":"not_applicable","study_design_consensus":null,"genre_codex":"review","genre_gemma":"review","genre_scores_codex":[0.00008715564,0.9988424,0.00009023547,0.0003667951,0.000132095,0.000001784248,0.000005816682,0.000005330387,0.0004684539],"genre_scores_gemma":[0.000464093,0.9988186,0.000131155,0.0001850073,0.00008743435,0.000002937676,0.000009160066,0.000001266974,0.0003003535],"genre_candidate":"review","genre_consensus":"review","teacher_disagreement_score":0.001973045,"threshold_uncertainty_score":0.00660044,"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."}}