{"id":"W2022040013","doi":"10.1073/pnas.0807027105","title":"Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics","year":2008,"lang":"en","type":"article","venue":"Proceedings of the National Academy of Sciences","topic":"Retinal Development and Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":664,"is_retracted":false,"has_abstract":true,"ca_institutions":"SickKids Foundation; Hospital for Sick Children; University of Toronto","funders":"National Eye Institute; National Heart, Lung, and Blood Institute","keywords":"RPE65; Cis-trans-Isomerases; Visual phototransduction; Retinoid; Genetic enhancement; Biology; Retinal; Retinal degeneration; Ophthalmology; Internal medicine; Gene; Endocrinology; Medicine; Chemistry; Isomerase; Biochemistry; Retinoic acid; Peptidylprolyl isomerase","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":[0.0001992785,0.0003167846,0.0002796793,0.0001657503,0.0001118513,0.0002801549,0.0001306514,0.0003225798,0.001167008],"category_scores_gemma":[0.0001522363,0.0001108373,0.0002561153,0.00009961093,0.0002724207,0.0001765011,0.00009818868,0.0005337076,0.0003681398],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0002531752,"about_ca_system_score_gemma":0.0001578856,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0005421718,"about_ca_topic_score_gemma":0.0005502926,"domain_scores_codex":[0.9998448,0.00002446073,0.00001451915,0.00003181167,0.00004815642,0.00003621567],"domain_scores_gemma":[0.9999001,0.00002351917,0.00003050366,0.00001919757,0.000009184978,0.00001748303],"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":[0.00008721081,0.00005776808,0.0001832979,0.00001536281,0.000003176957,0.0000460444,0.000016284,0.0000376796,0.9977853,0.0001053518,0.00003588654,0.001626536],"study_design_scores_gemma":[0.0000534421,0.003095233,0.004126121,0.000007431395,0.00002218977,0.0009783045,0.00002548494,0.0008720639,0.9873854,0.00006516471,0.003360664,0.000008404724],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9948211,0.00117353,0.002610345,0.00006796769,0.00002204198,0.000050251,0.000104793,0.0001062123,0.0010437],"genre_scores_gemma":[0.9956241,0.0005129468,0.00177058,0.00003240685,0.00000590938,0.00004877541,0.0001863909,0.00001921658,0.001799611],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.001167008,"threshold_uncertainty_score":0.003903985,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.02218262974502444,"score_gpt":0.285830316987306,"score_spread":0.2636476872422816,"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."}}