{"id":"W4414719674","doi":"10.1007/978-1-0716-4738-7_1","title":"DG9-Conjugated Morpholino-Based Exon 51-Skipping Therapy for Duchenne Muscular Dystrophy","year":2025,"lang":"en","type":"article","venue":"Methods in molecular biology","topic":"Muscle Physiology and Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":0,"is_retracted":false,"has_abstract":false,"ca_institutions":"Muscular Dystrophy Canada; University of Alberta","funders":"","keywords":"Duchenne muscular dystrophy; Exon skipping; Dystrophin; Exon; Morpholino; Genetic enhancement; Muscular dystrophy","routes":{"ca_aff":true,"ca_fund":false,"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":[0.0001296755,0.0002522413,0.0002991947,0.0001764336,0.0001747598,0.0001941389,0.0002213837,0.0004183532,0.001220752],"category_scores_gemma":[0.00006389595,0.0001033047,0.0001657923,0.0000899306,0.0002533667,0.0001260108,0.0001826942,0.0005032488,0.0002882281],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0002521928,"about_ca_system_score_gemma":0.0001874177,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0005345048,"about_ca_topic_score_gemma":0.0009527666,"domain_scores_codex":[0.9998854,0.00001727132,0.000011901,0.00003219824,0.00002932716,0.00002388252],"domain_scores_gemma":[0.999974,0.000005104053,0.000008294545,0.000003747876,0.000002443916,0.000006323748],"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":[0.0004364501,0.0001180515,0.000178161,0.0001163134,0.00001618488,0.0003939582,0.00001806525,0.0004410805,0.9761813,0.0005366764,0.0004532264,0.02111051],"study_design_scores_gemma":[0.0002650056,0.002239238,0.003799091,0.00003611815,0.0001033488,0.002716107,0.00002830759,0.002481581,0.9653975,0.0002694307,0.0226383,0.00002599504],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"methods","genre_scores_codex":[0.969658,0.009039485,0.01154583,0.0006144036,0.0003480918,0.0001262018,0.000504994,0.0004449787,0.007718064],"genre_scores_gemma":[0.9874905,0.002911405,0.003984809,0.000181031,0.00001998078,0.00004297645,0.0002957701,0.00003070595,0.005042773],"genre_candidate":"methods","genre_consensus":null,"teacher_disagreement_score":0.001220752,"threshold_uncertainty_score":0.004083872,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.01604644504800791,"score_gpt":0.3811811319172502,"score_spread":0.3651346868692423,"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."}}