{"id":"W2419441553","doi":"","title":"Juvenile GM2 Gangliosidosis: A Model for Investigation of Small-molecule Therapies for Lysosomal Storage Diseases","year":2008,"lang":"en","type":"dissertation","venue":"TSpace","topic":"Lysosomal Storage Disorders Research","field":"Medicine","cited_by":0,"is_retracted":false,"has_abstract":true,"ca_institutions":"","funders":"Hospital for Sick Children","keywords":"Gangliosidosis; Substrate reduction therapy; Juvenile; Medicine; Lysosomal storage disease; Neuroscience; Psychology; Biology; Internal medicine; Genetics; Enzyme replacement therapy; Disease","routes":{"ca_aff":false,"ca_fund":true,"ca_venue":false,"about_ca":false,"invisible_to_affiliation_only":true},"retraction":null,"screen":null,"direct_labels":[],"prediction":{"model_version":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0003663418,0.0006242061,0.0006943647,0.0003662291,0.0002802211,0.0003396861,0.0003968118,0.0004437579,0.0009599372],"category_scores_gemma":[0.000132093,0.0001547064,0.0003676502,0.0002815762,0.0002702294,0.0003415899,0.0002511688,0.0008168463,0.0002801792],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0004734638,"about_ca_system_score_gemma":0.0005677923,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.001418965,"about_ca_topic_score_gemma":0.002870408,"domain_scores_codex":[0.9998381,0.00004030782,0.0000115107,0.00003056834,0.00004037184,0.00003904403],"domain_scores_gemma":[0.999885,0.00001549617,0.00002702289,0.00001642451,0.000009979123,0.00004606988],"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":[0.002342735,0.0006338691,0.0009368657,0.000247773,0.00002985637,0.0005575611,0.00004563816,0.0003938842,0.985246,0.0006081967,0.0005814644,0.008376143],"study_design_scores_gemma":[0.003395543,0.06694933,0.04374294,0.0001758907,0.000580602,0.007625805,0.0002556802,0.006322684,0.8084678,0.001097972,0.06127904,0.000106681],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9726791,0.01502562,0.00528364,0.0008035073,0.000211697,0.0006090797,0.001622514,0.0001787441,0.003586172],"genre_scores_gemma":[0.9734621,0.01183829,0.009174949,0.0002645805,0.0001053795,0.000463292,0.001337357,0.0000349119,0.003319171],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.001418965,"threshold_uncertainty_score":0.003435254,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.04600101841907003,"score_gpt":0.3435286742449099,"score_spread":0.2975276558258399,"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."}}