{"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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.002574877,0.0006769016,0.001863724,0.0004917024,0.000143105,0.0005287943,0.0004784653,0.0006901822,0.001965655],"category_scores_gemma":[0.002389609,0.0002962592,0.001932609,0.0003170946,0.0003313879,0.0005762392,0.0003534715,0.0006764631,0.0002088305],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0006520784,"about_ca_system_score_gemma":0.0003818035,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00035142,"about_ca_topic_score_gemma":0.0009493975,"domain_scores_codex":[0.9983228,0.001089474,0.0001461658,0.0002064804,0.0001521687,0.00008291268],"domain_scores_gemma":[0.9979047,0.0008753695,0.0007579417,0.0001474111,0.0001089157,0.0002057731],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"randomized_trial","study_design_gemma":"observational","study_design_scores_codex":[0.8876336,0.003128674,0.02688023,0.001483202,0.01136686,0.0001087411,0.0001004833,0.0008164008,0.00924706,0.0001086294,0.0003353689,0.05879068],"study_design_scores_gemma":[0.2432701,0.5857023,0.1475621,0.0002261882,0.01501392,0.0004607431,0.0001172801,0.00113359,0.004123515,0.0002589282,0.002072248,0.00005923113],"study_design_candidate":"observational","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9904716,0.007622837,0.0002768417,0.00007708887,0.00004306038,0.0002788245,0.000362773,0.00001628337,0.000850728],"genre_scores_gemma":[0.99722,0.001307462,0.0003337751,0.00009956319,0.00007098867,0.0002298593,0.0004203789,0.000003409313,0.0003145899],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.002574877,"threshold_uncertainty_score":0.0136174,"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."}}