{"id":"W2087185853","doi":"10.1172/jci34316","title":"Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice","year":2008,"lang":"en","type":"article","venue":"Journal of Clinical Investigation","topic":"Virus-based gene therapy research","field":"Biochemistry, Genetics and Molecular Biology","cited_by":284,"is_retracted":false,"has_abstract":true,"ca_institutions":"","funders":"Institute of Genetics; James N. Kirby Foundation; National Eye Institute; National Institutes of Health; Research to Prevent Blindness; F. M. Kirby Foundation","keywords":"ABCA4; Biology; Genetic enhancement; Transduction (biophysics); Gene delivery; Retinal degeneration; Gene; Capsid; Adeno-associated virus; Retinitis pigmentosa; Vector (molecular biology); Viral vector; Virology; Genetics; Recombinant DNA","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":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.003953314,0.0001154298,0.00038656,0.0002352995,0.0000224947,0.00000577106,0.0001897643,0.0002620156,0.000002718922],"category_scores_gemma":[0.002103273,0.0001101304,0.0001252579,0.0002825667,0.0001214695,0.00001948491,0.00005753574,0.0004827705,0.000001892751],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0000860965,"about_ca_system_score_gemma":0.0003655797,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0001109498,"about_ca_topic_score_gemma":0.00233426,"domain_scores_codex":[0.9967653,0.001077838,0.001384152,0.0002182557,0.0003244998,0.0002299853],"domain_scores_gemma":[0.9986346,0.0002439258,0.0006473311,0.0001364667,0.000234907,0.0001027282],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"bench_or_experimental","study_design_gemma":"observational","study_design_scores_codex":[0.003297607,0.0002428046,0.4716313,0.000008810786,0.00003808876,0.00005141007,0.0001778899,0.001252163,0.5224999,7.81748e-7,0.00004065185,0.0007586103],"study_design_scores_gemma":[0.004094998,0.001751345,0.5057146,0.00008984148,0.000005834565,0.000007764419,0.000037156,0.0002507028,0.4878703,0.00008401856,0.00001289081,0.00008050532],"study_design_candidate":"bench_or_experimental","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.998678,0.0007646109,0.00001419308,0.000179637,0.0001237829,0.0002081037,0.00001267533,0.000001951161,0.00001707908],"genre_scores_gemma":[0.9985024,0.0009195108,0.0002028351,0.0001560468,0.0001517738,0.000004931435,0.0000266965,0.00001487913,0.00002092778],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.03462952,"threshold_uncertainty_score":0.4490988,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.04523876992661468,"score_gpt":0.365887957769594,"score_spread":0.3206491878429793,"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."}}