{"id":"W4316506432","doi":"10.1016/j.omtn.2023.01.006","title":"Sustained therapeutic benefits by transient reduction of TDP-43 using ENA-modified antisense oligonucleotides in ALS/FTD mice","year":2023,"lang":"en","type":"article","venue":"Molecular Therapy — Nucleic Acids","topic":"Amyotrophic Lateral Sclerosis Research","field":"Medicine","cited_by":27,"is_retracted":false,"has_abstract":true,"ca_institutions":"","funders":"Japan Science and Technology Agency; National Center of Neurology and Psychiatry; Precursory Research for Embryonic Science and Technology; Otsuka Pharmaceutical; Japan Society for the Promotion of Science; Ministry of Education, Culture, Sports, Science and Technology; Human Frontier Science Program; Nihon Medi-Physics; Japan Agency for Medical Research and Development; Japan Society for the Promotion of Science London; Université Laval","keywords":"Amyotrophic lateral sclerosis; Neurodegeneration; Frontotemporal dementia; Nucleic acid; Oligonucleotide; Systemic administration; In vivo; Medicine; Chemistry; Cytoplasm; Pharmacology; Neuroscience; Cell biology; Cancer research; Biology; Disease; Dementia; Pathology; Biochemistry","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":[0.000236768,0.0008463378,0.0005563322,0.0004251913,0.0001660963,0.0003184003,0.0003281629,0.0006870352,0.0007065251],"category_scores_gemma":[0.0001104808,0.000193448,0.0003818587,0.0001439707,0.0003611305,0.0002804732,0.0001499329,0.0009597982,0.0002003167],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0002936703,"about_ca_system_score_gemma":0.0002624004,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0007390991,"about_ca_topic_score_gemma":0.001003138,"domain_scores_codex":[0.9998057,0.00002182971,0.00002825492,0.00005806741,0.00004127023,0.00004484765],"domain_scores_gemma":[0.9998699,0.00001595554,0.00005229616,0.00001002239,0.00001470304,0.00003711905],"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":[0.000114651,0.00006644069,0.0000317036,0.00003130703,0.000006646645,0.00002756984,0.000009898157,0.00008232428,0.9987391,0.00006991578,0.00001943021,0.000800985],"study_design_scores_gemma":[0.00007505812,0.001729159,0.0008215328,0.000008647566,0.00005307121,0.0001087601,0.00001772273,0.0009738223,0.9947971,0.00007194428,0.001334999,0.000008275886],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9897574,0.00175244,0.005893773,0.0001822739,0.0001144614,0.000121163,0.0006107279,0.0004313478,0.0011365],"genre_scores_gemma":[0.9872944,0.00175506,0.006799161,0.0001167294,0.00003360682,0.000205222,0.0005021468,0.00005425395,0.003239457],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.0008463378,"threshold_uncertainty_score":0.002363563,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.03795346494441981,"score_gpt":0.3050348720937128,"score_spread":0.267081407149293,"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."}}