{"id":"W4401713439","doi":"10.1093/brain/awae266","title":"Exon 1-targeting miRNA reduces the pathogenic exon 1 HTT protein in Huntington's disease models","year":2024,"lang":"en","type":"article","venue":"Brain","topic":"Genetic Neurodegenerative Diseases","field":"Neuroscience","cited_by":21,"is_retracted":false,"has_abstract":true,"ca_institutions":"University of British Columbia","funders":"CHDI Foundation","keywords":"Exon; Huntington's disease; microRNA; Huntingtin; Biology; Disease; Genetics; Medicine; Gene; Pathology","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":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0004328726,0.0002444948,0.0001503148,0.0001395593,0.0001939272,0.0002756118,0.0004689368,0.00003928307,0.00006272999],"category_scores_gemma":[0.001818686,0.0001837843,0.0001331528,0.0005901659,0.0001418674,0.0002595228,0.0001781991,0.0002550114,0.0001218473],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000040509,"about_ca_system_score_gemma":0.0001906419,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00001373761,"about_ca_topic_score_gemma":0.000008528831,"domain_scores_codex":[0.9972968,0.0006314769,0.0003146976,0.0008430173,0.0004395148,0.0004744962],"domain_scores_gemma":[0.9989721,0.0003931677,0.00006329526,0.0003883552,0.00002382921,0.0001592083],"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":[0.00002878652,0.00006885086,0.00005734272,0.00005515793,0.000002097947,0.00009316438,0.0004548618,0.001427221,0.9911632,0.002463646,0.001517144,0.002668527],"study_design_scores_gemma":[0.0001940787,0.00003582326,0.001208034,0.0001704468,0.00001337374,0.000002674101,0.0001384557,0.02488684,0.9640052,0.006602524,0.002442558,0.0003000381],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9907868,0.0008949787,0.0001168572,0.006135724,0.0002865367,0.000919454,0.00005976838,0.000174816,0.0006250541],"genre_scores_gemma":[0.996703,0.00002435639,0.0001405544,0.001142911,0.0001975097,0.0003276012,0.000007469474,0.00005579068,0.001400767],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.02715805,"threshold_uncertainty_score":0.7494506,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.03476718477471152,"score_gpt":0.2704770933438143,"score_spread":0.2357099085691028,"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."}}