{"id":"W4389340916","doi":"10.1016/j.omtm.2023.101168","title":"Intrathecal delivery of a bicistronic AAV9 vector expressing β-hexosaminidase A corrects Sandhoff disease in a murine model: A dosage study","year":2023,"lang":"en","type":"article","venue":"Molecular Therapy — Methods & Clinical Development","topic":"Lysosomal Storage Disorders Research","field":"Medicine","cited_by":8,"is_retracted":false,"has_abstract":true,"ca_institutions":"Queen's University","funders":"Canadian Glycomics Network; Cyrus Tang Foundation","keywords":"Sandhoff disease; Vector (molecular biology); Intrathecal; Biology; Hexosaminidase; Viral vector; Anatomy; Virology; Cell biology; Medicine; Genetics; Recombinant DNA; Biochemistry; Surgery; Gene","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":[0.0005974709,0.0009286732,0.0007176942,0.0008674493,0.0002838812,0.0004352993,0.0006551897,0.0007857505,0.000867251],"category_scores_gemma":[0.0002413216,0.0002813664,0.0004267457,0.0002696725,0.0006968639,0.0006455046,0.0002011422,0.00132077,0.0003826675],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000610517,"about_ca_system_score_gemma":0.0004305669,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00116074,"about_ca_topic_score_gemma":0.00161371,"domain_scores_codex":[0.9994255,0.0001005003,0.00006874926,0.0001572857,0.0001412505,0.0001066386],"domain_scores_gemma":[0.9997216,0.00003656674,0.00008384718,0.00004081804,0.00003470114,0.00008239238],"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":[0.0002755749,0.0004583379,0.00008962421,0.00004735628,0.00001001202,0.00004450425,0.00002271026,0.0001545733,0.9969056,0.00007164687,0.00005258806,0.001867483],"study_design_scores_gemma":[0.0002151275,0.01590757,0.002055213,0.00001742948,0.00009947772,0.0003742978,0.00004774828,0.001770684,0.9777133,0.00006138992,0.00172102,0.00001667312],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9908665,0.001309934,0.005660518,0.0001747648,0.00007733274,0.0002534782,0.0004659644,0.0001519035,0.001039576],"genre_scores_gemma":[0.9842765,0.00198889,0.007634717,0.0001206081,0.00005657832,0.0002665918,0.0008119614,0.00006147121,0.004782696],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.00116074,"threshold_uncertainty_score":0.004429638,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.1307000670046294,"score_gpt":0.4789954004824443,"score_spread":0.3482953334778149,"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."}}