{"id":"W2575606794","doi":"10.1182/blood.v106.11.1285.1285","title":"Long-Term and Sustained Correction of the α-Galactosidase A Deficiency in Fabry Mice and Patient Cells Receiving Lentivirally Transduced Hematopoietic Stem/Progenitor Cells.","year":2005,"lang":"en","type":"article","venue":"Blood","topic":"Virus-based gene therapy research","field":"Biochemistry, Genetics and Molecular Biology","cited_by":3,"is_retracted":false,"has_abstract":true,"ca_institutions":"University Health Network; University of Toronto; Ontario Institute for Cancer Research","funders":"","keywords":"Progenitor cell; Haematopoiesis; Genetic enhancement; Stem cell; Viral vector; Context (archaeology); Transplantation; Biology; Bone marrow; Gene delivery; Ex vivo; Fabry disease; Immunology; Cancer research; Cell biology; Medicine; Internal medicine; In vivo; Disease; Recombinant DNA; Gene; Genetics","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":[0.0002767362,0.0002491546,0.0002500523,0.0001789049,0.0001326676,0.0002914298,0.0001999496,0.0004115502,0.0008572441],"category_scores_gemma":[0.0001177806,0.00008831227,0.0002019967,0.0001317894,0.0002096635,0.0001734818,0.0001291772,0.0005437606,0.0003151709],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0001532719,"about_ca_system_score_gemma":0.0001324791,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.000314331,"about_ca_topic_score_gemma":0.0002929628,"domain_scores_codex":[0.9998749,0.00001951397,0.00001439495,0.00002791609,0.00003160096,0.0000316424],"domain_scores_gemma":[0.9999148,0.0000156001,0.00001973351,0.00001618251,0.000007481858,0.00002618825],"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":[0.0001285598,0.00007965219,0.0002176487,0.0000181731,0.000004329534,0.0001181505,0.0000274154,0.00007497081,0.997542,0.0001179409,0.00004010319,0.001631078],"study_design_scores_gemma":[0.00005932623,0.002089546,0.004469673,0.000009354002,0.0000394183,0.001404407,0.00003134243,0.001453959,0.9875084,0.00007222009,0.002856034,0.000006343828],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9963679,0.0008198837,0.001919334,0.00004188191,0.00002218174,0.00003294674,0.0002228677,0.00006652125,0.0005062958],"genre_scores_gemma":[0.9963226,0.0003315382,0.001092358,0.00003063615,0.000006541221,0.00003125622,0.0003838148,0.00001763494,0.00178364],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.0008572441,"threshold_uncertainty_score":0.002867758,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.006903911366305191,"score_gpt":0.2306584486392391,"score_spread":0.223754537272934,"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."}}