{"id":"W2400001500","doi":"10.1089/hum.2016.013","title":"Novel Vector Design and Hexosaminidase Variant Enabling Self-Complementary Adeno-Associated Virus for the Treatment of Tay-Sachs Disease","year":2016,"lang":"en","type":"article","venue":"Human Gene Therapy","topic":"Virus-based gene therapy research","field":"Biochemistry, Genetics and Molecular Biology","cited_by":44,"is_retracted":false,"has_abstract":true,"ca_institutions":"University of Toronto; University of Manitoba; SickKids Foundation; Queen's University","funders":"National Institute of Environmental Health Sciences; Research Manitoba; National Cancer Institute; Kyowa Hakko Kirin; Research to Prevent Blindness","keywords":"Sandhoff disease; HEXA; Tay-Sachs disease; Gangliosidosis; Biology; Hexosaminidase; Protein subunit; Lysosomal storage disease; Genetics; Molecular biology; Gene; Medicine; Biochemistry; Disease; Internal medicine; Enzyme","routes":{"ca_aff":true,"ca_fund":true,"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":[0.000286243,0.000458472,0.000361653,0.0002595893,0.000152591,0.0005057281,0.0004486091,0.0004184868,0.0007040026],"category_scores_gemma":[0.0001571805,0.0001925562,0.0002900779,0.000227305,0.0002225876,0.0003113265,0.0002141128,0.000581986,0.0003962893],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0004066337,"about_ca_system_score_gemma":0.0003537565,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0006426123,"about_ca_topic_score_gemma":0.0007149251,"domain_scores_codex":[0.9997773,0.00003201659,0.00003139614,0.00005600822,0.00006929349,0.00003418076],"domain_scores_gemma":[0.9999043,0.00001062767,0.00003372654,0.000008102364,0.00001832383,0.00002485538],"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":[0.00007989486,0.00007848964,0.0001159585,0.00007940886,0.00001226889,0.00009967449,0.00003290689,0.0003859893,0.9919221,0.0008337584,0.00009675368,0.006262716],"study_design_scores_gemma":[0.00008086335,0.00117811,0.0004848438,0.00001068848,0.00006156959,0.0004120541,0.00002431686,0.003147423,0.983146,0.0001000819,0.01133738,0.00001661893],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9017536,0.004173988,0.0870866,0.000364484,0.0003179265,0.000815859,0.0006924492,0.0004741782,0.004320908],"genre_scores_gemma":[0.8862858,0.002379473,0.1045137,0.0001445308,0.00004038286,0.0004009962,0.001010015,0.00007243655,0.005152758],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.0007040026,"threshold_uncertainty_score":0.00295037,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.05894145234049301,"score_gpt":0.3180341877105298,"score_spread":0.2590927353700368,"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."}}