{"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":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0004229015,0.0002502858,0.0002191787,0.00005520487,0.000281459,0.00002703691,0.0002599649,0.00007191776,0.00008969444],"category_scores_gemma":[0.00001802864,0.0001483869,0.0001442317,0.00006728848,0.000120413,0.000005374395,0.00003520224,0.00003051163,0.000001869904],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00006647287,"about_ca_system_score_gemma":0.0001346609,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00005369405,"about_ca_topic_score_gemma":0.00009248966,"domain_scores_codex":[0.9985695,0.0001689652,0.0002648073,0.0004247887,0.0001859949,0.0003859208],"domain_scores_gemma":[0.9989406,0.0001616445,0.0001235273,0.0005315727,0.0001045766,0.0001381055],"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":[0.004058148,0.0008552037,0.0001377555,0.000006518259,0.0006897093,0.000004171832,0.0002430491,0.00003047377,0.9808337,0.00001994833,0.0001752597,0.01294605],"study_design_scores_gemma":[0.007695388,0.01029808,0.002143119,0.00001243875,0.00007208459,0.000003591199,0.00002845825,0.0001290904,0.9735227,0.0001537592,0.005694713,0.0002465873],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9812924,0.007356429,0.009156327,0.0003678554,0.00005603338,0.001397331,0.000347014,0.00002328115,0.000003315276],"genre_scores_gemma":[0.9894388,0.008091653,0.001139048,0.0002299787,0.0002827795,0.0003733237,0.0001272746,0.00006616765,0.0002509846],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.01269947,"threshold_uncertainty_score":0.605104,"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."}}