{"id":"W4408854527","doi":"10.1038/s41434-025-00524-x","title":"Optimization of adeno-associated viral (AAV) gene therapies vectors for balancing efficacy, longevity and safety for clinical application","year":2025,"lang":"en","type":"article","venue":"Gene Therapy","topic":"Virus-based gene therapy research","field":"Biochemistry, Genetics and Molecular Biology","cited_by":8,"is_retracted":false,"has_abstract":false,"ca_institutions":"National Research Council Canada; BC Children's Hospital; McGill University; University of British Columbia","funders":"National Research Council Canada; Canadian Institutes of Health Research; Michael Smith Health Research BC; Government of Canada","keywords":"Adeno-associated virus; Genetic enhancement; Longevity; Biology; Virology; Gene transfer; Gene delivery; Vector (molecular biology); Gene; Genetics; Recombinant DNA","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":[0.001120534,0.0007819337,0.0005309501,0.0005904917,0.000285598,0.001137964,0.0003531841,0.0003744266,0.0007646391],"category_scores_gemma":[0.0008491202,0.00027955,0.0003345158,0.0005091648,0.0004249178,0.0005746646,0.0003307097,0.0006467738,0.0003384345],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0009087512,"about_ca_system_score_gemma":0.0008877326,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0007077853,"about_ca_topic_score_gemma":0.001599815,"domain_scores_codex":[0.9992046,0.0001567542,0.00009420645,0.0001166287,0.0003435358,0.00008436924],"domain_scores_gemma":[0.9996678,0.00008465463,0.0001292197,0.00002103711,0.00006241875,0.00003494862],"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":[0.0001241401,0.00009136009,0.0002801541,0.00007164838,0.00001379126,0.00002200957,0.00002741234,0.002132208,0.9891053,0.0009744054,0.00007166198,0.007085943],"study_design_scores_gemma":[0.00003266105,0.0006816536,0.001157475,0.00001814432,0.00006132773,0.0001182807,0.00003455444,0.007854649,0.9853338,0.0003886055,0.004294093,0.00002474006],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.8804265,0.005192401,0.1093671,0.0002646608,0.0001047851,0.0004983773,0.0004490155,0.0003544545,0.003342745],"genre_scores_gemma":[0.914468,0.002873516,0.0796086,0.00007024559,0.00002628027,0.0002990874,0.0004488996,0.0001534958,0.002051901],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.001137964,"threshold_uncertainty_score":0.006593466,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.02440312465991572,"score_gpt":0.3565630768798135,"score_spread":0.3321599522198977,"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."}}