{"id":"W4322503763","doi":"10.3390/pharmaceutics15030778","title":"Recent Trends in Antisense Therapies for Duchenne Muscular Dystrophy","year":2023,"lang":"en","type":"review","venue":"Pharmaceutics","topic":"Muscle Physiology and Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":49,"is_retracted":false,"has_abstract":true,"ca_institutions":"University of Alberta","funders":"Defeat Duchenne Canada; Canadian Institutes of Health Research; Muscular Dystrophy Canada; University of Alberta; Alberta Innovates - Health Solutions; Women and Children's Health Research Institute; Children's Health Research Institute; Alberta Innovates; U.S. Department of Defense","keywords":"Duchenne muscular dystrophy; Antisense therapy; Exon skipping; Muscular dystrophy; Medicine; Genetic enhancement; Disease; Clinical trial; Dystrophin; Drug development; Bioinformatics; Exon; Drug; Gene; Biology; Genetics; Pharmacology; Internal medicine; Alternative splicing","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":[0.0002325483,0.000381565,0.0007393403,0.000199037,0.00006239214,0.00001505309,0.0002709396,0.0004001077,0.00003901961],"category_scores_gemma":[0.00005620826,0.0003415428,0.0004581572,0.0003357737,0.00008178724,0.000002190757,0.0001093241,0.0002422773,0.00002403108],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00002765193,"about_ca_system_score_gemma":0.00009743067,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.000004026141,"about_ca_topic_score_gemma":0.000016776,"domain_scores_codex":[0.998396,0.0001617871,0.0004086595,0.0005323915,0.00008076598,0.0004203469],"domain_scores_gemma":[0.9992778,0.00004652447,0.0001633368,0.0003809833,0.00005225362,0.00007908788],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"design_other","study_design_gemma":"not_applicable","study_design_scores_codex":[0.00006713351,0.00009204545,0.000002967637,0.002578503,0.000390094,0.000007686517,0.00002295112,0.00001267554,0.0006078026,0.00004087612,0.002563126,0.9936141],"study_design_scores_gemma":[0.0006509445,0.00009379288,0.000006728099,0.0003070301,0.0003171349,0.000005065516,0.00001336197,0.0000233653,0.0001571316,0.00007733719,0.9979843,0.0003637881],"study_design_candidate":"not_applicable","study_design_consensus":null,"genre_codex":"review","genre_gemma":"review","genre_scores_codex":[0.0004604261,0.9981213,0.00002594841,0.00007567042,0.0004776021,0.0005148415,0.0001967642,0.00002854562,0.00009889873],"genre_scores_gemma":[0.0001192155,0.9966412,0.0001118654,0.0001496206,0.0003477326,0.0002639973,0.001824568,0.0000957611,0.000446054],"genre_candidate":"review","genre_consensus":"review","teacher_disagreement_score":0.9954212,"threshold_uncertainty_score":0.9999037,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.1528171404274656,"score_gpt":0.4427749429702708,"score_spread":0.2899578025428052,"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."}}