{"id":"W4405617743","doi":"10.1001/jamaophthalmol.2024.5375","title":"Five-Year Outcomes of Lenadogene Nolparvovec Gene Therapy in Leber Hereditary Optic Neuropathy","year":2024,"lang":"en","type":"letter","venue":"JAMA Ophthalmology","topic":"Mitochondrial Function and Pathology","field":"Biochemistry, Genetics and Molecular Biology","cited_by":23,"is_retracted":false,"has_abstract":true,"ca_institutions":"University of Ottawa","funders":"NIHR Cambridge Biomedical Research Centre; Medical Research Council; School of Medicine, Emory University; Ministero della Salute; Moorfields Eye Hospital NHS Foundation Trust; National Institute for Health and Care Research; Bundesministerium für Bildung und Forschung; Moorfields Eye Charity; Sight Research UK; Agence Nationale de la Recherche; E-Rare; National Institutes of Health; Isaac Newton Trust; Emory University; Research to Prevent Blindness","keywords":"Medicine; Optic neuropathy; Leber's hereditary optic neuropathy; Genetic enhancement; Ophthalmology; Optic nerve; Gene; Genetics","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":[0.001845282,0.0002619217,0.0005639841,0.0003695593,0.0002750514,0.0004820723,0.0002737402,0.0005495696,0.0009390755],"category_scores_gemma":[0.001827291,0.00006958744,0.001196912,0.0002365547,0.0002732896,0.0004848077,0.000296442,0.0005471892,0.0001195344],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0007043677,"about_ca_system_score_gemma":0.0004551558,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.001169952,"about_ca_topic_score_gemma":0.002307125,"domain_scores_codex":[0.9995017,0.0001717692,0.00006087891,0.0000659935,0.0001183536,0.00008136715],"domain_scores_gemma":[0.9983158,0.0002747643,0.0008348741,0.0001033662,0.000152475,0.0003187832],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"observational","study_design_gemma":"nonrandomized_trial","study_design_scores_codex":[0.2127915,0.01198508,0.5435304,0.001242593,0.005212211,0.001187815,0.0005185725,0.00475396,0.01066895,0.0004118858,0.002940563,0.2047566],"study_design_scores_gemma":[0.006458162,0.1480967,0.833881,0.0002132206,0.002998696,0.0008558637,0.0002420778,0.00213643,0.002597863,0.0002745936,0.00216949,0.00007599627],"study_design_candidate":"nonrandomized_trial","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9956473,0.002690412,0.000164502,0.00007931482,0.0000286742,0.00005478574,0.000433918,0.00001031441,0.0008908286],"genre_scores_gemma":[0.9986089,0.0003205244,0.0000764946,0.00007436977,0.0000219176,0.0000292791,0.0005635253,0.000001462053,0.0003036592],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.001845282,"threshold_uncertainty_score":0.00975889,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.02042478219812265,"score_gpt":0.2647838837542291,"score_spread":0.2443591015561065,"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."}}