{"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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0007241626,0.0006840788,0.0005965297,0.0007864595,0.0002632639,0.0006527188,0.000331748,0.0007609359,0.002317752],"category_scores_gemma":[0.0003290673,0.0002274071,0.0004400612,0.0002390478,0.0008127456,0.0006598139,0.000298862,0.001335033,0.0006091659],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0003918333,"about_ca_system_score_gemma":0.0004194,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0006289536,"about_ca_topic_score_gemma":0.001214182,"domain_scores_codex":[0.999441,0.0001325886,0.00004428406,0.0001097137,0.0001940892,0.00007836177],"domain_scores_gemma":[0.9996918,0.00007921995,0.00009196241,0.0000636396,0.00002733153,0.00004608321],"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":[0.0001296948,0.0001127011,0.00007398117,0.00007079274,0.000008530481,0.00007487186,0.00003186387,0.0001000926,0.9962607,0.0003508369,0.0001145972,0.002671337],"study_design_scores_gemma":[0.00004908704,0.002145956,0.001108432,0.00002562667,0.00003052917,0.0007246177,0.00002700914,0.001077574,0.9890867,0.0001569959,0.005555619,0.0000118423],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9121777,0.005718794,0.07066612,0.0007343406,0.000291484,0.0007282143,0.0008081318,0.001069489,0.007805551],"genre_scores_gemma":[0.9554443,0.004014943,0.02926355,0.0001400691,0.00003691273,0.0004719836,0.0006736729,0.0001695966,0.009784942],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.002317752,"threshold_uncertainty_score":0.00775367,"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."}}