{"id":"W4207017208","doi":"10.1038/s41434-022-00316-7","title":"Antisense oligonucleotides as a potential treatment for brain deficits observed in myotonic dystrophy type 1","year":2022,"lang":"en","type":"article","venue":"Gene Therapy","topic":"Genetic Neurodegenerative Diseases","field":"Neuroscience","cited_by":45,"is_retracted":false,"has_abstract":true,"ca_institutions":"Institut Universitaire en Santé Mentale de Québec; Université Laval","funders":"U.S. Army Medical Research Acquisition Activity; Canadian Institutes of Health Research; AFM-Téléthon; Ionis Pharmaceuticals; French Muscular Dystrophy Association; Servier; Muscular Dystrophy Association; Government of Canada; U.S. Department of Defense","keywords":"Myotonic dystrophy; Myotonia; Systemic administration; Biology; Medicine; Atrophy; Neuroscience; Bioinformatics; Internal medicine; Endocrinology; In vivo; Genetics","routes":{"ca_aff":true,"ca_fund":true,"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":[0.0000986527,0.0002287937,0.000225495,0.0001267191,0.0003075245,0.00004749429,0.0002744215,0.00002389146,0.0002970501],"category_scores_gemma":[0.00008688407,0.0002189617,0.0001408062,0.0003824257,0.00005328702,0.00004358458,0.0000688048,0.00007238262,0.00003737633],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000115356,"about_ca_system_score_gemma":0.0001784046,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00006136489,"about_ca_topic_score_gemma":0.00001323083,"domain_scores_codex":[0.9980669,0.0004234213,0.0002286837,0.0006124392,0.0002583691,0.0004101893],"domain_scores_gemma":[0.9992951,0.0001374547,0.0000825314,0.0003644518,0.00003288328,0.00008757413],"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":[0.0009182229,0.0004208375,0.0002357431,0.000002829443,0.00001083283,0.00009044381,0.0002458789,0.008972594,0.9854506,0.0002013532,0.000180086,0.003270553],"study_design_scores_gemma":[0.00274097,0.002540607,0.003798463,0.000001805824,0.000008862092,0.00003321275,0.00006610413,0.0008119794,0.9861912,0.0005971428,0.002989253,0.0002204674],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9968348,0.000597334,0.00002663497,0.0007014977,0.0004349728,0.001143568,0.000153139,0.00006405504,0.00004404576],"genre_scores_gemma":[0.9960574,0.000361878,0.0002892902,0.00209143,0.0000708976,0.0003908462,0.00002232152,0.00005558534,0.0006603497],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.008160614,"threshold_uncertainty_score":0.8928998,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.0727699569777682,"score_gpt":0.2963358746395767,"score_spread":0.2235659176618085,"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."}}