{"id":"W1985262278","doi":"10.1038/mt.2014.6","title":"Gene Therapy Using a Liver-targeted AAV Vector Restores Nucleoside and Nucleotide Homeostasis in a Murine Model of MNGIE","year":2014,"lang":"en","type":"article","venue":"Molecular Therapy","topic":"Mitochondrial Function and Pathology","field":"Biochemistry, Genetics and Molecular Biology","cited_by":66,"is_retracted":false,"has_abstract":true,"ca_institutions":"","funders":"Institute of Genetics; National Institute of Neurological Disorders and Stroke; H2020 European Research Council; Eunice Kennedy Shriver National Institute of Child Health and Human Development; United Mitochondrial Disease Foundation; Instituto de Salud Carlos III; Fondazione Pierfranco e Luisa Mariani","keywords":"Thymidine phosphorylase; Biology; Mitochondrial DNA; Genetic enhancement; Cancer research; Virology; Gene; Molecular biology; Genetics; Cancer","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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0002585827,0.0007320449,0.0003786079,0.0008097293,0.000224896,0.000344463,0.0003924176,0.0008916822,0.000922533],"category_scores_gemma":[0.0001738414,0.0002441162,0.000348897,0.000160565,0.0006023716,0.0003701099,0.000263405,0.001032667,0.0003134132],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0004040803,"about_ca_system_score_gemma":0.0002908946,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0007508193,"about_ca_topic_score_gemma":0.001093173,"domain_scores_codex":[0.999729,0.00004421841,0.00003235452,0.00007054715,0.00006466552,0.00005921152],"domain_scores_gemma":[0.9997469,0.00003120724,0.00009971586,0.00002694793,0.00002065123,0.00007457341],"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":[0.000134091,0.00009549244,0.00008129937,0.00003968519,0.000005730333,0.00009726374,0.00002303153,0.0001299789,0.9979913,0.0001634974,0.0000399312,0.001198692],"study_design_scores_gemma":[0.0001080637,0.002842115,0.001786357,0.00003553257,0.00003684064,0.0006893988,0.0000461381,0.001948021,0.9889674,0.0001199673,0.003407872,0.00001230808],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9872455,0.001502027,0.008137221,0.0002521683,0.0001156521,0.0001703115,0.0004880731,0.0004424159,0.001646626],"genre_scores_gemma":[0.9834964,0.001444372,0.008003429,0.00009469301,0.0000300958,0.0001744678,0.0005802946,0.00006159236,0.006114572],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.000922533,"threshold_uncertainty_score":0.00308615,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.02484233677767201,"score_gpt":0.2493073379191438,"score_spread":0.2244650011414718,"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."}}