{"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":"codex-gemma-dda1882f352a","candidate_categories":["metaepi_narrow"],"consensus_categories":[],"category_scores_codex":[0.0001467347,0.000355347,0.000470577,0.0001241958,0.0007264017,0.00003219854,0.000520888,0.00003221913,0.0001958475],"category_scores_gemma":[0.00004019717,0.000276172,0.000323587,0.0003760938,0.0003049868,0.00005517656,0.00004472182,0.0001042991,0.00000312529],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00007903034,"about_ca_system_score_gemma":0.0001729593,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00005579502,"about_ca_topic_score_gemma":0.00000515439,"domain_scores_codex":[0.9974911,0.0004154037,0.0005841899,0.0006513405,0.0004211747,0.0004368269],"domain_scores_gemma":[0.9985167,0.0001840894,0.0003945459,0.0006431866,0.0001744014,0.00008707523],"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":[0.001372916,0.0009127121,0.00002028478,0.00002863088,0.00007671469,0.00001823628,0.0009911642,0.002212576,0.9928434,0.0002260595,0.00003548218,0.00126178],"study_design_scores_gemma":[0.002738087,0.00593286,0.0006311654,0.00001226908,0.00006335038,0.000004612127,0.000131128,0.0003130949,0.9889855,0.0001335998,0.0008125347,0.0002417531],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9939997,0.002464217,0.00006351522,0.00008998822,0.0002313015,0.002779597,0.0002861522,0.0000599759,0.00002558702],"genre_scores_gemma":[0.9974988,0.001050883,0.0002881012,0.0001455306,0.00007186535,0.0006759251,0.0000246818,0.00008026832,0.0001639842],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.005020148,"threshold_uncertainty_score":0.9999691,"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."}}