{"id":"W2800131432","doi":"10.1093/brain/awy126","title":"A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency","year":2018,"lang":"en","type":"article","venue":"Brain","topic":"Virus-based gene therapy research","field":"Biochemistry, Genetics and Molecular Biology","cited_by":127,"is_retracted":false,"has_abstract":true,"ca_institutions":"Université de Montréal; Centre Hospitalier Universitaire Sainte-Justine","funders":"Biotechnology and Biological Sciences Research Council; Medical Research Council; Moorfields Eye Hospital NHS Foundation Trust; National Institute for Health and Care Research; Vetenskapsrådet; Action Medical Research; Wellcome Trust; Kyowa Hakko Kirin; King's College London; European Commission; Pfizer","keywords":"Capsid; Adeno-associated virus; Virology; Transmembrane protein; Virus; Enzyme; Biology; Cell biology; Biochemistry; Recombinant DNA; Vector (molecular biology); Receptor; 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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0001742334,0.0005135547,0.0003167698,0.0002837059,0.0001726369,0.0003562628,0.0002801797,0.0004934589,0.0005965442],"category_scores_gemma":[0.0001969409,0.0001728446,0.0004027617,0.0001633868,0.0003360444,0.0002873046,0.00031663,0.0005242924,0.0002050808],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0004417329,"about_ca_system_score_gemma":0.0004899013,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.001250573,"about_ca_topic_score_gemma":0.00128194,"domain_scores_codex":[0.9998142,0.00001785048,0.00002188814,0.00004797544,0.00006571651,0.00003234688],"domain_scores_gemma":[0.9998512,0.00001624543,0.00005153594,0.00001225712,0.00001884999,0.0000498838],"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":[0.00006001216,0.00002994349,0.0001155448,0.00004615923,0.000007900318,0.0002290606,0.00001453234,0.0002105298,0.9968446,0.0002355009,0.00007361459,0.002132633],"study_design_scores_gemma":[0.0001615189,0.001533355,0.003207074,0.00002003432,0.00009138701,0.004099423,0.00002258775,0.003603084,0.9723498,0.00017731,0.0146973,0.000037263],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9334159,0.003050456,0.05825837,0.0003603759,0.0003232982,0.000243293,0.0006771525,0.001058742,0.002612399],"genre_scores_gemma":[0.9628229,0.001185384,0.03108993,0.0001216734,0.00004008524,0.00007405981,0.000914996,0.0001526157,0.003598411],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.001250573,"threshold_uncertainty_score":0.003205001,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.01418684951070015,"score_gpt":0.2678228293102372,"score_spread":0.2536359797995371,"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."}}