{"id":"W4411983578","doi":"10.1093/brain/awaf249","title":"Exon skipping peptide-conjugated morpholinos downregulate dynamin 2 to rescue centronuclear myopathy","year":2025,"lang":"en","type":"article","venue":"Brain","topic":"Cellular transport and secretion","field":"Biochemistry, Genetics and Molecular Biology","cited_by":4,"is_retracted":false,"has_abstract":true,"ca_institutions":"","funders":"Centre National de la Recherche Scientifique; Université de Strasbourg; Institute of Genetics; Institut National de la Santé et de la Recherche Médicale; Conseil National de la Recherche Scientifique; Agence Nationale de la Recherche","keywords":"Morpholino; Exon skipping; Dynamin; Exon; Biology; mdx mouse; Skeletal muscle; Molecular biology; Alternative splicing; Endocrinology; Cancer research; Gene knockdown; Dystrophin; Gene; Genetics; Receptor","routes":{"ca_aff":false,"ca_fund":true,"ca_venue":false,"about_ca":false,"invisible_to_affiliation_only":true},"retraction":null,"screen":null,"direct_labels":[],"prediction":{"model_version":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.000138362,0.0004751413,0.0002837919,0.0002476509,0.0001007345,0.0001757841,0.0002005189,0.0003674417,0.0008739837],"category_scores_gemma":[0.00006884265,0.0001499124,0.0002480891,0.00008526531,0.0002399134,0.0001860887,0.0001449161,0.0005961248,0.0002497354],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000186737,"about_ca_system_score_gemma":0.0002154147,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0004824143,"about_ca_topic_score_gemma":0.0009866515,"domain_scores_codex":[0.9998896,0.00001091287,0.00001296274,0.00003480243,0.00002936288,0.00002237108],"domain_scores_gemma":[0.9999199,0.00001020003,0.00003027972,0.000005564709,0.000008640517,0.00002551365],"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":[0.00004225145,0.00002320817,0.00003252058,0.00001982254,0.000002204579,0.00001841032,0.000004761404,0.00003310979,0.9993412,0.00003912625,0.00001712484,0.0004261651],"study_design_scores_gemma":[0.00004456158,0.0007407053,0.001410716,0.000006202704,0.0000178269,0.0001757746,0.00001494969,0.000601183,0.9948512,0.00003224595,0.002100069,0.000004598307],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9927365,0.001087336,0.004138883,0.0001252341,0.000066042,0.00007401067,0.0004494633,0.0002079663,0.001114645],"genre_scores_gemma":[0.9857569,0.001464603,0.005794131,0.0001403285,0.00002336889,0.0001152842,0.0006491047,0.00007449971,0.005981714],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.0008739837,"threshold_uncertainty_score":0.002923787,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.004502122533816184,"score_gpt":0.2208976541936715,"score_spread":0.2163955316598553,"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."}}