{"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":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0002607251,0.0001079757,0.0001198641,0.00006598813,0.00005987593,0.00002039381,0.00008882543,0.00006068356,0.00002812417],"category_scores_gemma":[0.0000342767,0.0000921475,0.00004134978,0.0001480857,0.00006091249,0.00000216959,0.00005184503,0.00004820813,0.00001661534],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00002777317,"about_ca_system_score_gemma":0.00004488561,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00007195297,"about_ca_topic_score_gemma":0.0005461904,"domain_scores_codex":[0.999137,0.0001339153,0.0001098861,0.0002590716,0.0001079682,0.0002520961],"domain_scores_gemma":[0.999654,0.00002634687,0.00001730808,0.000229151,0.00001815533,0.00005505438],"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":[0.0007785266,0.0000402743,0.01325897,0.0000104376,0.00007547106,0.00003637509,0.0004574084,0.00006063243,0.9653296,0.0000196226,0.0002655269,0.01966708],"study_design_scores_gemma":[0.004677814,0.004104177,0.06157165,0.00002150579,0.00001716208,0.00000941827,0.000724264,0.0004073136,0.8143268,0.0001619836,0.113562,0.0004159598],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9973477,0.001456439,0.00001726525,0.0006529513,0.00006307614,0.0002021859,0.00001075697,0.00002059248,0.0002290151],"genre_scores_gemma":[0.9952827,0.003219667,0.00001618587,0.0001087276,0.00008885641,0.00004080533,0.00006330806,0.00002177965,0.001157957],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.1510029,"threshold_uncertainty_score":0.3757666,"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."}}