{"id":"W4366352907","doi":"10.1038/s41434-023-00400-6","title":"rAAV-PHP.B escapes the mouse eye and causes lethality whereas rAAV9 can transduce aniridic corneal limbal stem cells without lethality","year":2023,"lang":"en","type":"article","venue":"Gene Therapy","topic":"Corneal Surgery and Treatments","field":"Medicine","cited_by":3,"is_retracted":false,"has_abstract":true,"ca_institutions":"BC Children's Hospital; University of British Columbia","funders":"Canadian Institutes of Health Research; British Columbia Knowledge Development Fund; Foundation Fighting Blindness; Government of Canada; Canada Foundation for Innovation; BC Children's Hospital","keywords":"Aniridia; PAX6; Gene delivery; Biology; Genetic enhancement; Transduction (biophysics); Adeno-associated virus; Virology; Ophthalmology; Medicine; Genetics; Gene; Recombinant DNA; Vector (molecular biology)","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":[0.0002887542,0.000682603,0.0002825244,0.0005389384,0.0001980342,0.0004042741,0.000385437,0.0006298891,0.001585074],"category_scores_gemma":[0.0001741012,0.000247722,0.0003684765,0.0001717488,0.0004862707,0.000297193,0.0002560462,0.001195255,0.0006843273],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000215023,"about_ca_system_score_gemma":0.0002477579,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0007131072,"about_ca_topic_score_gemma":0.0008886814,"domain_scores_codex":[0.9995572,0.0000708717,0.00004985016,0.0001295827,0.0001256892,0.00006685744],"domain_scores_gemma":[0.9995454,0.00009506143,0.0001688306,0.00008065213,0.00003202731,0.00007792041],"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":[0.00002385811,0.00001605154,0.0001292419,0.00002820752,0.000004483986,0.00005568272,0.00001170605,0.0000297462,0.9988092,0.0001114466,0.00001855074,0.0007617479],"study_design_scores_gemma":[0.00001133259,0.0004301859,0.00362181,0.00001201447,0.00001630053,0.0008839595,0.00002454717,0.0004853508,0.9924509,0.000077492,0.001980219,0.000005786846],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9743329,0.002472089,0.01699805,0.000174166,0.00004035942,0.00009410266,0.0009271989,0.0004670261,0.004494139],"genre_scores_gemma":[0.9788063,0.001645449,0.01086332,0.0001267266,0.00001180452,0.0001103968,0.001056321,0.0001681295,0.007211636],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.001585074,"threshold_uncertainty_score":0.005302548,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.04837754732226573,"score_gpt":0.2921559127128007,"score_spread":0.243778365390535,"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."}}