{"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":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0001668084,0.000166891,0.0002008774,0.00006614536,0.00004693025,0.00000774727,0.0001091252,0.0001317996,0.00002602611],"category_scores_gemma":[0.000001508829,0.000158188,0.00009416509,0.0001397125,0.0001261178,0.000002300584,0.0000198733,0.0000682235,0.000003007625],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00001558484,"about_ca_system_score_gemma":0.00004743982,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00007289453,"about_ca_topic_score_gemma":0.0000157549,"domain_scores_codex":[0.9987552,0.0001432225,0.0003266656,0.0003059146,0.0002485741,0.0002203831],"domain_scores_gemma":[0.9994175,0.00001596301,0.000153732,0.0002449405,0.0001196311,0.00004818322],"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":[0.001568204,0.0003248552,0.00006479999,0.00002256848,0.00005758941,4.395942e-7,0.0001762072,0.0009187114,0.9819649,3.768278e-7,0.0007635247,0.01413784],"study_design_scores_gemma":[0.001034631,0.002440825,0.00003457494,0.000008186207,0.00001023354,0.000003490159,0.0001203093,0.0003875414,0.9950212,0.000004927142,0.0007909193,0.0001431569],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9921451,0.006393848,0.0005838271,0.0000247696,0.0003274063,0.0003994889,0.00008353781,0.00001036182,0.00003164566],"genre_scores_gemma":[0.9987484,0.0008189331,0.00006344246,0.00002782657,0.00006863345,0.00002296326,0.00007681522,0.00002985843,0.0001431649],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.01399468,"threshold_uncertainty_score":0.6450719,"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."}}