{"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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0008166732,0.0006759285,0.0008985645,0.0005772078,0.0003751095,0.0008997678,0.0007181154,0.001059308,0.001124984],"category_scores_gemma":[0.0002690419,0.0003158995,0.0004943543,0.0002508343,0.0008926798,0.0007918198,0.000341522,0.001339805,0.0002660659],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000696525,"about_ca_system_score_gemma":0.0005154826,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0009729938,"about_ca_topic_score_gemma":0.001212365,"domain_scores_codex":[0.9996736,0.00007599169,0.0000244064,0.00008317553,0.00006735534,0.00007546219],"domain_scores_gemma":[0.9998816,0.0000251513,0.00003862062,0.00002078072,0.000008771187,0.0000249422],"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":[0.0007041403,0.0003002825,0.00008765187,0.00008752398,0.00002516461,0.0001765972,0.00008224948,0.0002876769,0.988686,0.001110803,0.0001384333,0.00831347],"study_design_scores_gemma":[0.0002138981,0.0009800637,0.0004929467,0.00001090951,0.00008078462,0.0005727327,0.00002925423,0.0009579172,0.9943885,0.0001866574,0.002073989,0.00001243372],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9666997,0.008606404,0.01980774,0.0005238517,0.0002849005,0.0002415916,0.0002346484,0.0002945545,0.003306673],"genre_scores_gemma":[0.9843149,0.002956778,0.009264766,0.00009436497,0.00005053711,0.00009709071,0.0001600323,0.0000530426,0.003008436],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.001124984,"threshold_uncertainty_score":0.005053639,"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."}}