{"id":"W2967945233","doi":"10.1089/hum.2019.132","title":"Dual <i>ABCA4</i> -AAV Vector Treatment Reduces Pathogenic Retinal A2E Accumulation in a Mouse Model of Autosomal Recessive Stargardt Disease","year":2019,"lang":"en","type":"article","venue":"Human Gene Therapy","topic":"Retinal Development and Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":58,"is_retracted":false,"has_abstract":true,"ca_institutions":"University of British Columbia","funders":"National Institutes of Health","keywords":"ABCA4; Stargardt disease; Biology; Retinal degeneration; Retinal; Retinal pigment epithelium; Transgene; Genetic enhancement; Cell biology; Molecular biology; Phenotype; Genetics; Gene; Biochemistry","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":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.00009541984,0.0001986723,0.0001907779,0.00006372845,0.00004835082,0.00001560242,0.0001211345,0.00008706842,0.00006648271],"category_scores_gemma":[0.000007149933,0.000176286,0.0001085658,0.00006056904,0.000043876,0.000008449381,0.00003266341,0.00004367608,0.000007955948],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00003556968,"about_ca_system_score_gemma":0.0001656741,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00001776402,"about_ca_topic_score_gemma":0.00002156306,"domain_scores_codex":[0.9989161,0.00007340327,0.0002683575,0.0003655134,0.0001578653,0.0002187291],"domain_scores_gemma":[0.9994547,0.000005957884,0.0001327596,0.0002785704,0.00005453484,0.00007343342],"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":[0.001141215,0.0002141571,0.0135174,0.000009604131,0.00004458299,0.000002455891,0.0003575139,0.001783804,0.9819991,0.00003804261,0.00005143717,0.000840674],"study_design_scores_gemma":[0.003310903,0.0009817265,0.03376259,0.0000179496,0.00001391638,0.000001211026,0.00008821367,0.0009278965,0.9598657,0.0003107551,0.0003708389,0.0003483216],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9981676,0.0009154433,0.00003424053,0.0000536235,0.00004354316,0.000418076,0.0000480138,0.00001258366,0.0003068395],"genre_scores_gemma":[0.9962915,0.0009274713,0.0003414582,0.00006506877,0.00006150656,0.00004645608,0.0004374564,0.00002815421,0.001800901],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.02213344,"threshold_uncertainty_score":0.7188734,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.02996646924036832,"score_gpt":0.2857423452787368,"score_spread":0.2557758760383685,"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."}}