{"id":"W4409365773","doi":"10.2147/dnnd.s495536","title":"Gene Editing for Duchenne Muscular Dystrophy: From Experimental Models to Emerging Therapies","year":2025,"lang":"en","type":"review","venue":"Degenerative Neurological and Neuromuscular Disease","topic":"CRISPR and Genetic Engineering","field":"Biochemistry, Genetics and Molecular Biology","cited_by":9,"is_retracted":false,"has_abstract":true,"ca_institutions":"Muscular Dystrophy Canada; University of Alberta","funders":"","keywords":"Duchenne muscular dystrophy; Genome editing; Muscular dystrophy; Medicine; Computer science; Computational biology; Gene; Biology; CRISPR; Genetics; Internal medicine","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":"codex-gemma-dda1882f352a","candidate_categories":["metaepi_narrow"],"consensus_categories":[],"category_scores_codex":[0.00009120735,0.0006843098,0.0009029832,0.0001057108,0.0002206348,0.00008742312,0.0003189106,0.0002444772,0.00001306336],"category_scores_gemma":[0.0001698704,0.0005590513,0.0006924617,0.0001362036,0.00006941044,0.000007746588,0.0004000465,0.0002049202,0.000002318299],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000009026277,"about_ca_system_score_gemma":0.00007474908,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.000004456216,"about_ca_topic_score_gemma":0.000001479134,"domain_scores_codex":[0.9973239,0.0002450029,0.0004311848,0.001374069,0.0001763767,0.000449507],"domain_scores_gemma":[0.9988356,0.0000788827,0.00009301048,0.0005073123,0.00007003381,0.0004151129],"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":[0.001189715,0.0012421,0.00004288654,0.009894799,0.002507398,0.0006438881,0.0003404394,0.01727535,0.1235574,0.0009713546,0.004475335,0.8378593],"study_design_scores_gemma":[0.0007901982,0.0009881951,0.00005400549,0.0007196895,0.001133955,0.00001179103,0.0000208249,0.001702282,0.007285572,0.0001618767,0.9858543,0.001277316],"study_design_candidate":"not_applicable","study_design_consensus":null,"genre_codex":"review","genre_gemma":"review","genre_scores_codex":[0.03046633,0.9510041,0.01581612,0.0001123506,0.0004049305,0.00135202,0.0007932438,0.00004161737,0.000009259749],"genre_scores_gemma":[0.008572847,0.984258,0.002136626,0.000981334,0.001352413,0.001290347,0.001186505,0.00009910712,0.0001228468],"genre_candidate":"review","genre_consensus":"review","teacher_disagreement_score":0.981379,"threshold_uncertainty_score":0.9996861,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.03109345762769829,"score_gpt":0.3319463459376829,"score_spread":0.3008528883099846,"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."}}