{"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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.00007380274,0.0002995524,0.0002990561,0.0002662331,0.0001183019,0.0001367432,0.0001589944,0.0002844214,0.00102598],"category_scores_gemma":[0.00005567115,0.00005240417,0.0001725651,0.0001329072,0.0001288485,0.0001098636,0.00009221592,0.0004203642,0.0001630407],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0001389682,"about_ca_system_score_gemma":0.000156589,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0004927618,"about_ca_topic_score_gemma":0.00109002,"domain_scores_codex":[0.9999689,0.000005728736,0.000003430913,0.000009301742,0.000006594351,0.00000612329],"domain_scores_gemma":[0.999985,0.000002946789,0.000004518878,9.044221e-7,0.000003077377,0.000003485825],"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":[0.0009464928,0.0002856562,0.000218471,0.0005431686,0.00004636528,0.0003093757,0.00005505656,0.0002906596,0.9241818,0.001170086,0.0007706668,0.07118219],"study_design_scores_gemma":[0.001279734,0.01894939,0.005979748,0.000207072,0.00047926,0.002439097,0.0001179438,0.00186461,0.8923317,0.000939674,0.07535724,0.00005459736],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9147958,0.06322061,0.01013868,0.0008730544,0.0006502373,0.0003327059,0.0005330236,0.0004446615,0.009011148],"genre_scores_gemma":[0.9384887,0.04506587,0.00741775,0.000327712,0.000149332,0.0002787226,0.0005265053,0.00002476452,0.007720625],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.00102598,"threshold_uncertainty_score":0.003432214,"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."}}