{"id":"W4389477769","doi":"10.1016/j.medj.2023.11.008","title":"Safety concerns surrounding AAV and CRISPR therapies in neuromuscular treatment","year":2023,"lang":"en","type":"article","venue":"Med","topic":"Virus-based gene therapy research","field":"Biochemistry, Genetics and Molecular Biology","cited_by":6,"is_retracted":false,"has_abstract":false,"ca_institutions":"University of Alberta","funders":"Neuroscience and Mental Health Institute, University of Alberta; Defeat Duchenne Canada; Muscular Dystrophy Canada; Alberta Innovates; Alberta Innovates - Health Solutions; Women and Children's Health Research Institute","keywords":"CRISPR; Duchenne muscular dystrophy; Transactivation; Neuromuscular disease; Genome editing; Medicine; Adeno-associated virus; Genetic enhancement; Bioinformatics; Biology; Virology; Gene; Genetics; Disease; Vector (molecular biology); Internal medicine","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":[0.0159128,0.0002584079,0.0005542046,0.0006366762,0.001040156,0.002437788,0.0009282389,0.00313781,0.004277426],"category_scores_gemma":[0.01299359,0.0001811885,0.0007341946,0.0002811331,0.002401216,0.001017651,0.0007688759,0.003086078,0.0009893309],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.001048819,"about_ca_system_score_gemma":0.001714894,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0007892058,"about_ca_topic_score_gemma":0.00184263,"domain_scores_codex":[0.9929827,0.003598955,0.0003307676,0.0004238606,0.002471873,0.0001918342],"domain_scores_gemma":[0.9867487,0.009870459,0.00134542,0.0005226577,0.001224713,0.0002879589],"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":[0.003025237,0.0003773648,0.01027378,0.002576345,0.0003959142,0.003883319,0.001798621,0.002797668,0.09317307,0.1121109,0.02757809,0.7420098],"study_design_scores_gemma":[0.0003712209,0.008604321,0.01465845,0.003292229,0.0005907867,0.03287822,0.002219963,0.005298979,0.12666,0.07163416,0.7336363,0.0001554257],"study_design_candidate":"not_applicable","study_design_consensus":null,"genre_codex":"review","genre_gemma":"review","genre_scores_codex":[0.1948268,0.2913894,0.1795719,0.1421093,0.007425651,0.0006080915,0.0004743152,0.001009843,0.1825847],"genre_scores_gemma":[0.8516979,0.0556931,0.03170315,0.0339462,0.003788723,0.0004046474,0.0001819344,0.0001820547,0.02240237],"genre_candidate":"review","genre_consensus":"review","teacher_disagreement_score":0.0159128,"threshold_uncertainty_score":0.08415592,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.04021001273589409,"score_gpt":0.3390050651565665,"score_spread":0.2987950524206724,"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."}}