{"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":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0001976404,0.0002080943,0.0002693985,0.000119004,0.00004580119,0.0000140445,0.0001466941,0.0001784066,0.00002626026],"category_scores_gemma":[0.00005185224,0.0001979374,0.0001030511,0.0001214725,0.00009775402,0.000005164653,0.00005193519,0.00009551906,0.000002150883],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0000118582,"about_ca_system_score_gemma":0.00004602825,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0001150275,"about_ca_topic_score_gemma":0.00003121645,"domain_scores_codex":[0.9987652,0.0002027332,0.0002604208,0.0004029137,0.0001230109,0.0002456828],"domain_scores_gemma":[0.9993296,0.00001480608,0.0001054745,0.0004059811,0.00006631801,0.00007783756],"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":[0.0005922787,0.0000900773,0.0006694333,0.000008322338,0.00004774687,0.0000111913,0.0001403006,0.001674454,0.990023,0.000119669,0.00003846242,0.006585083],"study_design_scores_gemma":[0.002254192,0.0005811429,0.001404113,0.00001199865,0.000008000754,0.00001995193,0.00001242887,0.008303198,0.9842115,0.0002315335,0.00271665,0.0002453096],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9816339,0.004663214,0.01316532,0.00006173136,0.00009956397,0.0002573469,0.00001638778,0.00001408423,0.00008847121],"genre_scores_gemma":[0.9876144,0.002683422,0.008730749,0.0007463127,0.00008185742,0.00001487188,0.00004056944,0.00004767672,0.0000401306],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.006628744,"threshold_uncertainty_score":0.8071653,"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."}}