{"id":"W2972310174","doi":"10.1016/j.ymthe.2019.09.010","title":"Non-viral Gene Therapy for Stargardt Disease with ECO/pRHO-ABCA4 Self-Assembled Nanoparticles","year":2019,"lang":"en","type":"article","venue":"Molecular Therapy","topic":"Retinal Development and Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":51,"is_retracted":false,"has_abstract":true,"ca_institutions":"","funders":"National Eye Institute; Harrington Discovery Institute, University Hospitals; Alcon Research Institute; National Institute of General Medical Sciences; Canadian Institute for Advanced Research; Foundation Fighting Blindness; U.S. Department of Veterans Affairs","keywords":"ABCA4; Genetic enhancement; Stargardt disease; Virology; Biology; Gene; Genetics","routes":{"ca_aff":false,"ca_fund":true,"ca_venue":false,"about_ca":false,"invisible_to_affiliation_only":true},"retraction":null,"screen":null,"direct_labels":[],"prediction":{"model_version":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0001381665,0.0002913765,0.0002064548,0.00004489986,0.00008838892,0.000059011,0.0002542197,0.0001001304,0.00004187146],"category_scores_gemma":[0.000005174812,0.0002310865,0.0001590469,0.00009094546,0.00002273529,0.000008774817,0.00003393668,0.00005561868,0.00004081229],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00001474699,"about_ca_system_score_gemma":0.0001869715,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00000484147,"about_ca_topic_score_gemma":0.000002552008,"domain_scores_codex":[0.998628,0.00004333704,0.0001897521,0.0005191244,0.0002044517,0.0004152991],"domain_scores_gemma":[0.9991852,0.000008059489,0.0000795879,0.0004698536,0.0001063521,0.000150924],"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":[0.002751064,0.0002091456,0.01694729,0.00001564959,0.0002336077,0.000009154405,0.00007073762,0.0001013652,0.9774206,0.00007434568,0.0002838142,0.001883176],"study_design_scores_gemma":[0.005260077,0.001310242,0.01181073,0.000009824226,0.00001648513,0.000002650784,0.00003641061,0.0001199056,0.9506639,0.0002740191,0.03003191,0.0004638167],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9944004,0.002053589,0.001586093,0.0002668916,0.0000982125,0.001144047,0.000007862729,0.00003911624,0.0004037173],"genre_scores_gemma":[0.9943812,0.0009281235,0.002418914,0.001390975,0.0000580676,0.0001982519,0.0001547155,0.00007608101,0.0003936649],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.0297481,"threshold_uncertainty_score":0.9423432,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.004141365515033051,"score_gpt":0.2165472848154669,"score_spread":0.2124059193004338,"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."}}