{"id":"W4386019635","doi":"10.1080/14737175.2023.2249607","title":"Gene therapy for Duchenne muscular dystrophy: an update on the latest clinical developments","year":2023,"lang":"en","type":"review","venue":"Expert Review of Neurotherapeutics","topic":"Muscle Physiology and Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":24,"is_retracted":false,"has_abstract":true,"ca_institutions":"Université Laval","funders":"Canadian Institutes of Health Research; Health Research","keywords":"Duchenne muscular dystrophy; Exon skipping; Medicine; Nonsense mutation; Neuromuscular disease; Dystrophin; Bioinformatics; Muscular dystrophy; Genetic enhancement; Disease; Genetics; Exon; Gene; Mutation; Biology; Internal medicine; Alternative splicing; Missense mutation","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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0006097782,0.0007208157,0.001059242,0.001562036,0.0001981959,0.0009090608,0.0007123415,0.001024125,0.005535028],"category_scores_gemma":[0.000753626,0.0002100783,0.0006703674,0.001279489,0.0003065811,0.001023652,0.0005009741,0.002082447,0.002416983],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0005199027,"about_ca_system_score_gemma":0.0007477807,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0007255999,"about_ca_topic_score_gemma":0.001596491,"domain_scores_codex":[0.9998115,0.00003424586,0.00002697181,0.00003174575,0.00007486906,0.00002082718],"domain_scores_gemma":[0.9996697,0.0001846285,0.00003391542,0.000007332815,0.00007989207,0.00002456482],"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":[0.00008685201,0.00007275827,0.00008952263,0.01660383,0.0000787094,0.0002113756,0.00005877598,0.0002887239,0.001865463,0.003235367,0.05979555,0.9176132],"study_design_scores_gemma":[0.00002407533,0.0001107383,0.0003091495,0.004354409,0.0001010967,0.0008922763,0.00002815038,0.00006151594,0.0002771537,0.001032523,0.9927968,0.00001211299],"study_design_candidate":"not_applicable","study_design_consensus":null,"genre_codex":"review","genre_gemma":"review","genre_scores_codex":[0.0001087064,0.9976667,0.0001517333,0.0004355011,0.0004082324,0.0000070513,0.00003147673,0.00001178258,0.001178789],"genre_scores_gemma":[0.00041765,0.998104,0.0001987156,0.0003810913,0.0001898586,0.000008837561,0.00004507326,0.000002479032,0.0006523175],"genre_candidate":"review","genre_consensus":"review","teacher_disagreement_score":0.005535028,"threshold_uncertainty_score":0.01851654,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.1716447845702138,"score_gpt":0.4577435667961549,"score_spread":0.2860987822259411,"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."}}