{"id":"W4399106479","doi":"10.1093/brain/awae168","title":"Failure of <i>C9orf72</i> sense repeat-targeting antisense oligonucleotides: lessons learned and the path forward","year":2024,"lang":"en","type":"letter","venue":"Brain","topic":"Neurogenetic and Muscular Disorders Research","field":"Medicine","cited_by":25,"is_retracted":false,"has_abstract":true,"ca_institutions":"","funders":"Alzheimer’s Research UK; UK Dementia Research Institute; Academy of Medical Sciences; Medical Research Council Canada; LifeArc","keywords":"Sense (electronics); C9orf72; Oligonucleotide; Path (computing); Disappointment; Computer science; Medicine; Biology; Psychology; Disease; Internal medicine; Genetics; Engineering; Computer network","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":[0.004888889,0.0006640463,0.00144464,0.0004644138,0.001694326,0.002019526,0.001394795,0.02672076,0.002095732],"category_scores_gemma":[0.01762651,0.0003742345,0.001233161,0.0004003593,0.003329933,0.003311195,0.0007415737,0.02895542,0.003211284],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.003102092,"about_ca_system_score_gemma":0.00181423,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.002440765,"about_ca_topic_score_gemma":0.003668235,"domain_scores_codex":[0.9973906,0.0008572551,0.000411502,0.0003515087,0.0007604317,0.0002286784],"domain_scores_gemma":[0.9859407,0.01030564,0.0005389049,0.0003738778,0.001905809,0.0009351302],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"not_applicable","study_design_gemma":"not_applicable","study_design_scores_codex":[0.0003521189,0.0001389554,0.00223902,0.0003186522,0.00006910699,0.01491288,0.0002814693,0.0004221424,0.002471762,0.009675047,0.8648004,0.1043185],"study_design_scores_gemma":[0.0005813133,0.0006245878,0.002069525,0.0006926364,0.00009885045,0.02774668,0.0006560051,0.001681358,0.003401841,0.04300437,0.9192882,0.0001545531],"study_design_candidate":"not_applicable","study_design_consensus":"not_applicable","genre_codex":"commentary","genre_gemma":"commentary","genre_scores_codex":[0.001017322,0.007103596,0.0004273487,0.9793591,0.01050155,0.000009684038,0.00003570502,0.00004241479,0.001503406],"genre_scores_gemma":[0.01674709,0.01188994,0.001329022,0.9028282,0.06363744,0.00004841023,0.00005748337,0.00004789527,0.003414602],"genre_candidate":"commentary","genre_consensus":"commentary","teacher_disagreement_score":0.02672076,"threshold_uncertainty_score":0.02585524,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.02402705301895439,"score_gpt":0.3078787008247608,"score_spread":0.2838516478058064,"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."}}