{"id":"W3198457279","doi":"10.1016/j.omtm.2021.09.002","title":"AAV-mediated PEX1 gene augmentation improves visual function in the PEX1-Gly844Asp mouse model for mild Zellweger spectrum disorder","year":2021,"lang":"en","type":"article","venue":"Molecular Therapy — Methods & Clinical Development","topic":"Peroxisome Proliferator-Activated Receptors","field":"Biochemistry, Genetics and Molecular Biology","cited_by":21,"is_retracted":false,"has_abstract":true,"ca_institutions":"McGill University; Université de Montréal; McGill University Health Centre","funders":"Canadian Institutes of Health Research; Fonds de Recherche du Québec - Santé; Waikato Medical Research Foundation; Global Foundation for Peroxisomal Disorders","keywords":"Genetic enhancement; Peroxisomal disorder; Zellweger syndrome; Biology; Retinal; Retinal degeneration; Adeno-associated virus; Medicine; Ophthalmology; Gene; Vector (molecular biology); Genetics; Peroxisome","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":["metaepi_narrow"],"consensus_categories":[],"category_scores_codex":[0.002448039,0.0004087847,0.000429822,0.0000899564,0.0001733614,0.00007846866,0.0003329744,0.0004569521,0.00002904071],"category_scores_gemma":[0.0004580864,0.0003302755,0.000310651,0.0003104982,0.00009748478,0.00001520971,0.0001283752,0.0003523029,0.00001010223],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00006860255,"about_ca_system_score_gemma":0.0005180253,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.000006443478,"about_ca_topic_score_gemma":0.00002323661,"domain_scores_codex":[0.9958351,0.001334669,0.0009920419,0.0009560334,0.0003448302,0.0005373524],"domain_scores_gemma":[0.9987202,0.0002219748,0.0002367397,0.000501575,0.0001791534,0.000140324],"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":[0.0007787463,0.0005821976,0.001224049,0.00001508388,0.0003225286,0.000003568119,0.0002492646,0.0003560748,0.9524553,0.00002141652,0.000130541,0.04386128],"study_design_scores_gemma":[0.003042395,0.0004492513,0.001839033,0.000008201348,0.0000319877,0.000003206961,0.0001525733,0.003620716,0.9720259,0.0003432124,0.01801862,0.000464879],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.6044866,0.0009771863,0.3930737,0.000525525,0.0002693596,0.0006182292,0.00001105757,0.00002150449,0.00001676167],"genre_scores_gemma":[0.6025044,0.002518844,0.380143,0.01024371,0.000334258,0.001095744,0.001919267,0.0001854771,0.00105528],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.0433964,"threshold_uncertainty_score":0.9999149,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.04984800186399824,"score_gpt":0.4034345776349497,"score_spread":0.3535865757709515,"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."}}