{"id":"W2561260190","doi":"10.1016/s1525-0016(16)34320-9","title":"711. Intravenous Neonatal Gene Therapy Corrects GM2 Gangliosidosis in Sandhoff Mice for Long-Term, By Using AAV Viral Vector Expressing a New Hexosaminidase Variant","year":2015,"lang":"en","type":"article","venue":"Molecular Therapy","topic":"Lysosomal Storage Disorders Research","field":"Medicine","cited_by":0,"is_retracted":false,"has_abstract":true,"ca_institutions":"Hospital for Sick Children; University of Toronto; University of Manitoba; Queen's University","funders":"","keywords":"Gangliosidosis; Sandhoff disease; Hexosaminidase; HEXA; Ganglioside; Activator (genetics); Biology; Genetic enhancement; Molecular biology; Enzyme; Internal medicine; Biochemistry; Medicine; 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":"codex-gemma-dda1882f352a","candidate_categories":["metaepi_narrow"],"consensus_categories":[],"category_scores_codex":[0.0003705198,0.0004169587,0.0005855867,0.0002660874,0.0001071271,0.0001320921,0.0003233044,0.0002406239,0.00007106634],"category_scores_gemma":[0.0001402576,0.0003821311,0.0002039069,0.0004320113,0.00009941215,0.000169705,0.00006123166,0.0003338123,0.00001232935],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0003614522,"about_ca_system_score_gemma":0.0006248006,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0008010391,"about_ca_topic_score_gemma":0.00002461199,"domain_scores_codex":[0.9972161,0.0002202346,0.0004121379,0.0006592856,0.0006743419,0.000817884],"domain_scores_gemma":[0.9985821,0.0000849337,0.0001268877,0.0005102908,0.0001965169,0.0004992551],"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":[0.004015128,0.0006727137,0.002162205,0.00004331,0.000207386,0.001989495,0.002844542,0.00006096209,0.8980018,0.000004668917,0.0005038763,0.08949395],"study_design_scores_gemma":[0.02926095,0.002751765,0.003378072,0.0002825875,0.00005313118,0.0005957481,0.0002381319,0.002746421,0.9582015,0.0001317735,0.001585603,0.0007742985],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9421541,0.02273393,0.03246262,0.0004993339,0.0002526993,0.001760353,0.00002680302,0.00007413146,0.00003601921],"genre_scores_gemma":[0.9933022,0.0003791557,0.003917315,0.001548218,0.0002504052,0.0001017141,0.0001694928,0.0001657093,0.0001658415],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.08871964,"threshold_uncertainty_score":0.9998631,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.04447656347538877,"score_gpt":0.3164683759677134,"score_spread":0.2719918124923247,"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."}}