{"id":"W4284691266","doi":"10.1089/hum.2022.069","title":"Enhanced Delivery of Ligand-Conjugated Antisense Oligonucleotides (C16-HA-ASO) Targeting Dystrophia Myotonica Protein Kinase Transcripts for the Treatment of Myotonic Dystrophy Type 1","year":2022,"lang":"en","type":"article","venue":"Human Gene Therapy","topic":"Genetic Neurodegenerative Diseases","field":"Neuroscience","cited_by":23,"is_retracted":false,"has_abstract":true,"ca_institutions":"Institut Universitaire en Santé Mentale de Québec; Université Laval","funders":"Canadian Institutes of Health Research","keywords":"Myotonic dystrophy; Mutant; Medicine; Molecular biology; Oligonucleotide; Gene; Biology; Internal medicine; Biochemistry","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":[0.0001384306,0.0004385386,0.0003593832,0.0001820884,0.00008441688,0.0002053369,0.0001910171,0.0003267057,0.0005627864],"category_scores_gemma":[0.0000536365,0.0001186404,0.0002005623,0.0001021375,0.0002023429,0.0001923386,0.0001490332,0.0004542588,0.0001634878],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0002429269,"about_ca_system_score_gemma":0.0002250474,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0005227223,"about_ca_topic_score_gemma":0.0008302674,"domain_scores_codex":[0.999891,0.00001485308,0.000008874078,0.00002738982,0.00003191119,0.00002593493],"domain_scores_gemma":[0.9999427,0.000006108921,0.00002409935,0.000002904153,0.000005743947,0.00001857241],"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":[0.00005514083,0.00002219603,0.0000274593,0.00002801994,0.000002875681,0.00001222605,0.000006181812,0.00006159693,0.99925,0.0000332606,0.00001112779,0.0004898435],"study_design_scores_gemma":[0.00004372836,0.001385187,0.0006934953,0.000004531456,0.00002306552,0.0001167917,0.00001245324,0.0007202805,0.9954822,0.00001270209,0.001500331,0.000005287093],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9946048,0.001480796,0.002587968,0.00005992587,0.00005334646,0.00005842514,0.000120841,0.00006312325,0.0009707811],"genre_scores_gemma":[0.9889466,0.001936981,0.005247819,0.00004302534,0.00001685112,0.00007152787,0.0002167623,0.00001598484,0.00350444],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.0005627864,"threshold_uncertainty_score":0.001882732,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.0465830261427314,"score_gpt":0.2767554449716011,"score_spread":0.2301724188288697,"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."}}