{"id":"W2021285531","doi":"10.1038/sj.gt.3302839","title":"Efficient correction of Fabry mice and patient cells mediated by lentiviral transduction of hematopoietic stem/progenitor cells","year":2006,"lang":"en","type":"article","venue":"Gene Therapy","topic":"Virus-based gene therapy research","field":"Biochemistry, Genetics and Molecular Biology","cited_by":55,"is_retracted":false,"has_abstract":false,"ca_institutions":"University Health Network; University of Toronto; Ontario Institute for Cancer Research","funders":"National Heart, Lung, and Blood Institute; National Institutes of Health","keywords":"CD34; Haematopoiesis; Progenitor cell; Biology; Bone marrow; Stem cell; Fabry disease; Globotriaosylceramide; Genetic enhancement; Viral vector; Transduction (biophysics); Immunology; Transplantation; Molecular biology; Cancer research; Cell biology; Internal medicine; Medicine; Recombinant DNA; Gene; Disease; Biochemistry","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":[0.0005373852,0.0005739746,0.000380022,0.0006092437,0.0002149328,0.0005552293,0.0003812216,0.0005724557,0.001336643],"category_scores_gemma":[0.0003921195,0.0002676683,0.0003041217,0.0002349606,0.0003835866,0.0002651952,0.0003739872,0.0008503813,0.0005644877],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0004063607,"about_ca_system_score_gemma":0.0004188098,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0006111606,"about_ca_topic_score_gemma":0.0006824398,"domain_scores_codex":[0.99959,0.00008053651,0.00004465653,0.0000606479,0.0001454702,0.00007869318],"domain_scores_gemma":[0.9997634,0.00006116829,0.0000686094,0.00005867415,0.00001844578,0.00002957764],"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":[0.00003347232,0.00001194836,0.0001061984,0.00001578476,0.000004466386,0.0001007144,0.00002724726,0.0001508683,0.9962445,0.001082916,0.00005453822,0.002167226],"study_design_scores_gemma":[0.00001109314,0.00004189723,0.0003628458,0.000003035127,0.00001075571,0.0005536593,0.000005755243,0.0009592681,0.9945239,0.00009627522,0.003429013,0.000002506234],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9194691,0.001842922,0.07195198,0.0003047764,0.0001300649,0.0002314108,0.0008147472,0.001260376,0.003994635],"genre_scores_gemma":[0.9794847,0.0006246778,0.01401109,0.00005171543,0.0000161809,0.00004723626,0.0005099109,0.0001937383,0.005060711],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.001336643,"threshold_uncertainty_score":0.004471481,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.007039856845197663,"score_gpt":0.2266742752517625,"score_spread":0.2196344184065648,"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."}}