{"id":"W4412697087","doi":"10.1007/978-1-0716-4730-1_8","title":"Direct Reprogramming of Human DMD Fibroblasts into Myotubes for In Vitro Evaluation of Antisense-Mediated Exon Skipping and Exons 45–55 Skipping Accompanied by Rescue of Dystrophin Expression","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":"Exon skipping; Reprogramming; Myogenesis; Exon; Dystrophin; Duchenne muscular dystrophy; In vitro; Cell biology; Biology; Molecular biology; Chemistry; Genetics; Gene; Alternative splicing","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":[0.0004152871,0.000413561,0.0003427145,0.0001591054,0.0002567684,0.000311445,0.0002158387,0.0003673596,0.001780589],"category_scores_gemma":[0.000171716,0.0002150901,0.0004182961,0.0001559868,0.0002210222,0.0001431502,0.0002193914,0.0005724287,0.0005651563],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0002367267,"about_ca_system_score_gemma":0.0002580138,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00153343,"about_ca_topic_score_gemma":0.002378389,"domain_scores_codex":[0.9996818,0.00004875749,0.00006158507,0.00007154289,0.00008700962,0.00004922978],"domain_scores_gemma":[0.9998509,0.00005347142,0.00002844782,0.0000280146,0.00001945709,0.0000197376],"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":[0.00002488176,0.00001208846,0.00004247718,0.00001381749,0.000002516026,0.00002818486,0.00002120102,0.00003267139,0.9994109,0.00006241306,0.00001248044,0.0003364531],"study_design_scores_gemma":[0.000007646329,0.0000987275,0.000945738,0.000001955142,0.00001126008,0.00009630749,0.00001626946,0.0003119411,0.9971854,0.00001532784,0.001307001,0.000002362065],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9736018,0.0009639442,0.01697232,0.0001360875,0.0001518166,0.0002628511,0.001986331,0.0001696578,0.005755359],"genre_scores_gemma":[0.9754561,0.0009555335,0.01107149,0.00009373436,0.00001607193,0.000206443,0.001707201,0.00009835266,0.01039501],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.001780589,"threshold_uncertainty_score":0.005956709,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.0203943565517919,"score_gpt":0.4026886095305712,"score_spread":0.3822942529787793,"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."}}