{"id":"W4415269065","doi":"10.1101/2025.10.15.682364","title":"Gene therapy-mediated overexpression of wild-type MFN2 improves Charcot-Marie-Tooth disease type 2A","year":2025,"lang":"en","type":"preprint","venue":"bioRxiv (Cold Spring Harbor Laboratory)","topic":"Hereditary Neurological Disorders","field":"Neuroscience","cited_by":0,"is_retracted":false,"has_abstract":true,"ca_institutions":"Canadian Nautical Research Society","funders":"École Polytechnique Fédérale de Lausanne; Institut National de la Santé et de la Recherche Médicale; Agence Nationale de la Recherche","keywords":"MFN2; Endoplasmic reticulum; Genetic enhancement; Mitochondrion; Transgene; Gene; Viral vector; Genetically modified mouse; mitochondrial fusion","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":[0.0002079693,0.0006913797,0.0005088952,0.0004667938,0.0001857629,0.0003891629,0.0002674466,0.0007438007,0.001539214],"category_scores_gemma":[0.0001172995,0.0001172525,0.0003597027,0.0001837839,0.0003448986,0.0003789933,0.0001913226,0.0009945333,0.0003821166],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0003178047,"about_ca_system_score_gemma":0.0002163812,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0006365479,"about_ca_topic_score_gemma":0.0009208635,"domain_scores_codex":[0.9998252,0.00002055615,0.00001826444,0.00004426148,0.0000574414,0.00003429204],"domain_scores_gemma":[0.9999268,0.000008939863,0.00002000486,0.000006102384,0.00001000435,0.00002807161],"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":[0.0002137173,0.0002179705,0.00005118378,0.00008034427,0.00001264388,0.00005525658,0.00001828017,0.0001270769,0.9961289,0.0001160821,0.0001610759,0.002817544],"study_design_scores_gemma":[0.0002066885,0.004311222,0.003428316,0.00003183911,0.00008841211,0.0006189124,0.00006004394,0.001966804,0.9795136,0.0001675609,0.009586309,0.00002033762],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9859833,0.00358521,0.005244994,0.0003364321,0.0003092308,0.0001299191,0.0007514672,0.0005273283,0.003132044],"genre_scores_gemma":[0.9883576,0.002339144,0.00317714,0.0001288798,0.00005103295,0.0001321771,0.0007022349,0.00006414299,0.005047738],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.001539214,"threshold_uncertainty_score":0.005149126,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.02446924599922424,"score_gpt":0.2408886673873906,"score_spread":0.2164194213881664,"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."}}