{"id":"W2012516911","doi":"10.1016/j.ymgme.2008.08.003","title":"In vivo delivery of human acid ceramidase via cord blood transplantation and direct injection of lentivirus as novel treatment approaches for Farber disease","year":2008,"lang":"en","type":"article","venue":"Molecular Genetics and Metabolism","topic":"Lysosomal Storage Disorders Research","field":"Medicine","cited_by":36,"is_retracted":false,"has_abstract":false,"ca_institutions":"Ontario Institute for Cancer Research; University of Toronto","funders":"National Institute of Neurological Disorders and Stroke; U.S. Public Health Service","keywords":"Genetic enhancement; Progenitor cell; Transgene; Severe combined immunodeficiency; In vivo; Viral vector; Ceramide; Transplantation; Cancer research; Haematopoiesis; CD34; Hematopoietic stem cell transplantation; Xenotransplantation; Biology; Immunology; Stem cell; Medicine; Internal medicine; Cell biology; Biochemistry; Gene; Recombinant DNA; Apoptosis","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":[0.00005616944,0.0001169707,0.0002569737,0.0001163943,0.00004205876,0.000004275806,0.00002509864,0.00005073376,0.000004981943],"category_scores_gemma":[0.0000113174,0.0001056416,0.00006846005,0.00009511141,0.00009956393,0.00002640581,0.00001248268,0.00003611117,9.115338e-8],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000009864563,"about_ca_system_score_gemma":0.00004197901,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0003908616,"about_ca_topic_score_gemma":0.00003227785,"domain_scores_codex":[0.9992802,0.00003066459,0.0001861181,0.0002085239,0.0001651159,0.0001293566],"domain_scores_gemma":[0.9996626,0.00001281539,0.00005212233,0.0001172714,0.00006247602,0.00009273444],"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":[0.0007073369,0.0008465921,0.005929752,0.0002479622,0.000158408,0.0000576383,0.0007033026,0.00005447567,0.9829367,0.0001494738,0.000001590776,0.008206747],"study_design_scores_gemma":[0.006739302,0.0007909935,0.0780553,0.00002712466,0.0006411547,0.00005673722,0.00004088076,0.0008527919,0.9123453,0.0001892295,0.0001387296,0.0001224134],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9838896,0.01306596,0.002189904,0.00002041054,0.0000192292,0.0006331066,0.00006492774,0.000004180343,0.0001127245],"genre_scores_gemma":[0.9958018,0.003235708,0.0007571726,0.00002094565,0.00001694676,0.00006010123,0.00002689379,0.00001727422,0.00006314719],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.07212555,"threshold_uncertainty_score":0.4307939,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.03705379081205669,"score_gpt":0.2816511345084585,"score_spread":0.2445973436964018,"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."}}