{"id":"W4402973519","doi":"10.1016/j.omtm.2024.101349","title":"Single cell and TCR analysis of immune cells from AAV gene therapy-dosed Duchenne muscular dystrophy patients","year":2024,"lang":"en","type":"article","venue":"Molecular Therapy — Methods & Clinical Development","topic":"Muscle Physiology and Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":10,"is_retracted":false,"has_abstract":true,"ca_institutions":"","funders":"American Lebanese Syrian Associated Charities; Petroleum Technology Alliance Canada; University of California, Los Angeles; National Institutes of Health; National Institute of Neurological Disorders and Stroke; Cystic Fibrosis Foundation Therapeutics; National Institute of Arthritis and Musculoskeletal and Skin Diseases; National Institute of Allergy and Infectious Diseases; National Heart, Lung, and Blood Institute; Pfizer; Cystic Fibrosis Foundation; U.S. Department of Defense","keywords":"Duchenne muscular dystrophy; Dystrophin; Peripheral blood mononuclear cell; Muscular dystrophy; Dosing; T-cell receptor; mdx mouse; Peripheral blood; Medicine; Immunology; Biology; T cell; Genetics; Internal medicine; Immune system","routes":{"ca_aff":false,"ca_fund":true,"ca_venue":false,"about_ca":false,"invisible_to_affiliation_only":true},"retraction":null,"screen":null,"direct_labels":[],"prediction":{"model_version":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0002370291,0.0002323284,0.0002905966,0.000821499,0.0003206353,0.0003651868,0.0001285231,0.0004541959,0.002032883],"category_scores_gemma":[0.0005164384,0.0001416901,0.0001971585,0.0002699902,0.000231836,0.000102237,0.0001758787,0.0003570295,0.0003947681],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0001633588,"about_ca_system_score_gemma":0.0000796323,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0007274616,"about_ca_topic_score_gemma":0.0007140423,"domain_scores_codex":[0.9997523,0.0000336536,0.00003131432,0.00009061756,0.00005614769,0.00003593836],"domain_scores_gemma":[0.9997644,0.0000604725,0.00003410147,0.00002311107,0.00005476887,0.00006312383],"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":[0.001243951,0.0002817686,0.1033705,0.00007735088,0.00006333263,0.00842716,0.001310009,0.0002048153,0.8666162,0.0001109026,0.0003313542,0.01796274],"study_design_scores_gemma":[0.0001785468,0.00645848,0.7038528,0.00005802116,0.000181938,0.05345645,0.001471358,0.002730723,0.2262029,0.0002408387,0.005127512,0.00004051004],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9973813,0.0002216569,0.001139996,0.00002952884,0.00001073568,0.00003862392,0.0002808304,0.00002048493,0.0008769236],"genre_scores_gemma":[0.9975731,0.0001281906,0.0008134036,0.00009863844,0.000009498925,0.00004532604,0.0004044964,0.00001459421,0.0009128748],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.002032883,"threshold_uncertainty_score":0.006800652,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.02836669504461219,"score_gpt":0.3428925713759017,"score_spread":0.3145258763312895,"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."}}