{"id":"W4310465424","doi":"10.1007/s40123-022-00611-x","title":"Indirect Comparison of Lenadogene Nolparvovec Gene Therapy Versus Natural History in Patients with Leber Hereditary Optic Neuropathy Carrying the m.11778G>A MT-ND4 Mutation","year":2022,"lang":"en","type":"article","venue":"Ophthalmology and Therapy","topic":"Retinal Development and Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":30,"is_retracted":false,"has_abstract":true,"ca_institutions":"University of Ottawa","funders":"National Institute of Neurological Disorders and Stroke; National Eye Institute; NIHR Cambridge Biomedical Research Centre; National Institute for Health Research Biomedical Research Centre at Moorfields Eye Hospital NHS Foundation Trust and UCL Institute of Ophthalmology; UCL Institute of Ophthalmology, University College London; GENSIGHT BIOLOGICS PARIS; Università di Bologna; Medical Research Council; School of Medicine, Emory University; Moorfields Eye Charity; Sight Research UK; National Institutes of Health; Isaac Newton Trust; Ministero della Salute; Bundesministerium für Bildung und Forschung; National Institute for Health and Care Research; Agence Nationale de la Recherche; Emory University; University College London; Vlaamse regering; E-Rare; Moorfields Eye Hospital NHS Foundation Trust; Universiteit Gent; University of Colorado School of Medicine, Anschutz Medical Campus; Thomas Jefferson University; Icahn School of Medicine at Mount Sinai; Chiesi Farmaceutici; Massachusetts Eye and Ear; Bijzonder Onderzoeksfonds UGent; Fight for Sight UK","keywords":"Natural history; Optic neuropathy; Medicine; Visual acuity; Leber's hereditary optic neuropathy; Genetic enhancement; Mutation; Ophthalmology; Gene; Internal medicine; Optic nerve; Genetics; Biology","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":[0.0001612631,0.0001629374,0.0002032274,0.00006548507,0.0001378817,0.000004585782,0.0001687871,0.00006878871,0.0000678086],"category_scores_gemma":[0.000009895717,0.000122687,0.00004425932,0.00009245878,0.0001494098,0.000007292547,0.00005885709,0.0001774575,6.124293e-7],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00003235859,"about_ca_system_score_gemma":0.00007681599,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00004310647,"about_ca_topic_score_gemma":0.0000063316,"domain_scores_codex":[0.9989318,0.0002337307,0.0002022187,0.0002855545,0.0001558503,0.0001908383],"domain_scores_gemma":[0.9995801,0.00003936571,0.0001358444,0.000179083,0.00004066608,0.00002491523],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"observational","study_design_gemma":"observational","study_design_scores_codex":[0.01754267,0.0007265985,0.9054248,0.0000149722,0.0003525784,0.0001375791,0.002196765,0.0007049144,0.05834357,0.00002531125,0.000455469,0.01407478],"study_design_scores_gemma":[0.01030115,0.005091422,0.961825,0.000006714296,0.00001969755,0.0002152259,0.0005209034,0.0001159326,0.01585956,0.00004522254,0.005538471,0.0004607401],"study_design_candidate":"observational","study_design_consensus":"observational","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9810755,0.0176175,0.00000223521,0.00005799917,0.0005180131,0.000275779,0.00000606233,0.000004549606,0.0004424083],"genre_scores_gemma":[0.9992096,0.0002263139,0.00007212763,0.0001132632,0.00003115326,0.00004822242,0.0002024868,0.00002008609,0.00007677476],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.05640017,"threshold_uncertainty_score":0.500303,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.01733473439479705,"score_gpt":0.2415158198863837,"score_spread":0.2241810854915866,"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."}}