{"id":"W2577036545","doi":"10.1182/blood.v114.22.3576.3576","title":"Generation and Implementation of a Novel Murine Xenograft Model for Evaluating Human Hematopoietic Cell-Targeted Gene Therapies for Fabry Disease.","year":2009,"lang":"en","type":"article","venue":"Blood","topic":"Lysosomal Storage Disorders Research","field":"Medicine","cited_by":0,"is_retracted":false,"has_abstract":true,"ca_institutions":"Hamilton Health Sciences; University Health Network; Ontario Institute for Cancer Research","funders":"","keywords":"Severe combined immunodeficiency; Nod; Globotriaosylceramide; Fabry disease; Biology; Spleen; Transplantation; Sandhoff disease; Krabbe disease; Genetic enhancement; Immunology; Internal medicine; Medicine; Endocrinology; In vivo; Gene; Disease; Leukodystrophy; Diabetes mellitus; 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":[0.00112955,0.0006656258,0.000435983,0.0006009489,0.0002956038,0.000609759,0.0005297241,0.0008995634,0.003112511],"category_scores_gemma":[0.0002750294,0.0003329288,0.0005142338,0.0002913648,0.0002781357,0.0006297597,0.0002817597,0.001342163,0.001242788],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0003466106,"about_ca_system_score_gemma":0.0004318029,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.001132203,"about_ca_topic_score_gemma":0.00106103,"domain_scores_codex":[0.9995993,0.00008544554,0.0000416312,0.00009289331,0.0001102912,0.00007050861],"domain_scores_gemma":[0.9996362,0.00005946667,0.00007125406,0.00008808365,0.00005352043,0.00009144351],"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":[0.0005808606,0.0007640051,0.0002455422,0.00008062717,0.00001739066,0.0001502485,0.00005552364,0.0004911596,0.9914732,0.0007004339,0.0007160646,0.004724848],"study_design_scores_gemma":[0.0002743884,0.007307738,0.001971507,0.00003457822,0.00009487583,0.0008200236,0.00007156415,0.004586227,0.9694439,0.0001437277,0.01523201,0.0000195399],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.8716502,0.004593687,0.09762344,0.0007432092,0.001192836,0.002935937,0.00773109,0.001434904,0.01209464],"genre_scores_gemma":[0.8924745,0.003526004,0.07464374,0.000276133,0.0001341832,0.002228026,0.00733086,0.0003392321,0.01904734],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.003112511,"threshold_uncertainty_score":0.01041245,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.0885928812565905,"score_gpt":0.3976911981508325,"score_spread":0.309098316894242,"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."}}