{"id":"W2055335510","doi":"10.1002/iub.284","title":"Therapy for lysosomal storage disorders","year":2009,"lang":"en","type":"review","venue":"IUBMB Life","topic":"Lysosomal Storage Disorders Research","field":"Medicine","cited_by":138,"is_retracted":false,"has_abstract":true,"ca_institutions":"Tellabs (Canada)","funders":"","keywords":"Lysosomal storage disorders; Substrate reduction therapy; Enzyme replacement therapy; Lysosomal storage disease; Genetic enhancement; Orphan drug; Medicine; Disease; Fabry disease; Hematopoietic stem cell transplantation; Intensive care medicine; Bioinformatics; Internal medicine; Biology; Gene","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":[0.0003429714,0.0007232067,0.00106967,0.001820961,0.0002768261,0.0004836049,0.0005812581,0.0007854743,0.004165402],"category_scores_gemma":[0.0002887198,0.000112472,0.0004675203,0.0009113902,0.0003415715,0.0005527791,0.0005358437,0.0009818452,0.003922777],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0003009897,"about_ca_system_score_gemma":0.0003044072,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0003440923,"about_ca_topic_score_gemma":0.0007134301,"domain_scores_codex":[0.9998544,0.00002998942,0.00001600972,0.00002566237,0.00006109819,0.00001279966],"domain_scores_gemma":[0.9999357,0.00002517752,0.00001184087,0.000005221086,0.00001454997,0.000007510439],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"design_other","study_design_gemma":"not_applicable","study_design_scores_codex":[0.0001022772,0.0001427179,0.000152847,0.007488371,0.0000912604,0.0008366874,0.00009206673,0.0002240165,0.01560914,0.003171724,0.02135698,0.9507319],"study_design_scores_gemma":[0.00006368745,0.0002493462,0.0007358267,0.001599536,0.00009743161,0.007582709,0.00005249659,0.00009396815,0.006081943,0.001696012,0.9817286,0.00001845458],"study_design_candidate":"not_applicable","study_design_consensus":null,"genre_codex":"review","genre_gemma":"review","genre_scores_codex":[0.0006631055,0.9942757,0.0007014548,0.0002483475,0.0002446966,0.00001583767,0.0000283449,0.00002783223,0.003794615],"genre_scores_gemma":[0.00433257,0.9896322,0.001138627,0.0004653414,0.0002053444,0.00002770323,0.00009461407,0.000006968208,0.004096526],"genre_candidate":"review","genre_consensus":"review","teacher_disagreement_score":0.004165402,"threshold_uncertainty_score":0.01393467,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.07941979709710534,"score_gpt":0.3984812059653671,"score_spread":0.3190614088682618,"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."}}