{"id":"W4408865117","doi":"10.1126/sciadv.adt9354","title":"Retinal gene therapy for Stargardt disease with dual AAV intein vectors is both safe and effective in large animal models","year":2025,"lang":"en","type":"article","venue":"Science Advances","topic":"Retinal Development and Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":15,"is_retracted":false,"has_abstract":true,"ca_institutions":"University of British Columbia","funders":"","keywords":"Stargardt disease; Intein; Genetic enhancement; Transduction (biophysics); Biology; Retinal; Gene; Genetics; RNA","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":[0.0002456577,0.0001074498,0.00009193735,0.00007161479,0.0001327568,0.00003174079,0.0001199137,0.00002344243,0.000002593645],"category_scores_gemma":[0.00004943079,0.00008141276,0.00002082079,0.0002303552,0.0002916412,0.00003173817,0.00005610373,0.00003543643,2.380291e-7],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0000144049,"about_ca_system_score_gemma":0.000205476,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.000004791179,"about_ca_topic_score_gemma":0.00003336436,"domain_scores_codex":[0.9991236,0.00001650701,0.00008960534,0.0003986901,0.0001218972,0.0002496682],"domain_scores_gemma":[0.9997336,0.00001475286,0.00003217926,0.0001020595,0.00005816276,0.00005918993],"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":[0.0100488,0.000265829,0.3075737,0.0001143411,0.00004495156,0.000009986938,0.0008540402,0.000423507,0.6448488,0.002127847,0.0003086079,0.03337958],"study_design_scores_gemma":[0.004376519,0.002240546,0.3178375,0.0001377504,0.00001687817,0.000002660591,0.001063013,0.001259658,0.648182,0.006871236,0.01737778,0.0006344261],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9916481,0.003559367,0.003644801,0.0002344205,0.00004422506,0.0003852945,0.00001797828,0.000004869841,0.0004609755],"genre_scores_gemma":[0.9972584,0.0004507172,0.001738485,0.0002975362,0.00001430859,0.00007100493,0.00001186356,0.000004787325,0.0001528895],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.03274516,"threshold_uncertainty_score":0.3319916,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.00454986176629647,"score_gpt":0.2668583879383613,"score_spread":0.2623085261720648,"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."}}