{"id":"W2156696931","doi":"10.1371/journal.pone.0006810","title":"MECP2 Isoform-Specific Vectors with Regulated Expression for Rett Syndrome Gene Therapy","year":2009,"lang":"en","type":"article","venue":"PLoS ONE","topic":"Genetics and Neurodevelopmental Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":78,"is_retracted":false,"has_abstract":true,"ca_institutions":"University of Toronto; Hospital for Sick Children; SickKids Foundation","funders":"Natural Sciences and Engineering Research Council of Canada; Canadian Institutes of Health Research; Hospital for Sick Children; National Science Council; International Rett Syndrome Foundation","keywords":"MECP2; Rett syndrome; Biology; Viral vector; Gene isoform; Gene silencing; Ectopic expression; Cell biology; Molecular biology; Genetics; Gene; Phenotype","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":[0.00005003144,0.0001689271,0.0001554718,0.00003573779,0.00009567451,0.00002083097,0.0001488699,0.0001038734,0.0000213137],"category_scores_gemma":[0.000007039111,0.0001396142,0.00004719888,0.00008187122,0.0000298377,0.000003265721,0.00002284287,0.00005073175,0.000004689902],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000008759685,"about_ca_system_score_gemma":0.00002620714,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":7.500147e-7,"about_ca_topic_score_gemma":0.000001215682,"domain_scores_codex":[0.9990543,0.00001603877,0.0001499054,0.0003592675,0.000169586,0.0002509683],"domain_scores_gemma":[0.9995012,0.000004395361,0.00005906042,0.0002955856,0.00006760776,0.00007210609],"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":[0.0003212088,0.0004675728,0.0001804344,0.000008287273,0.00006213551,0.000002260662,0.00003215028,0.000009437837,0.9975675,0.000005713022,0.0005343914,0.0008088925],"study_design_scores_gemma":[0.001043653,0.001662962,0.03271309,0.00003740734,0.00001138465,0.000007797003,0.00001343191,0.00001089837,0.9623526,0.00009019315,0.001834965,0.0002215791],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9978839,0.0009595114,0.0002325118,0.0002171836,0.00002384895,0.0004730774,0.00001384036,0.00002254872,0.00017358],"genre_scores_gemma":[0.9914701,0.001268713,0.006104964,0.0003336976,0.00002421758,0.00004131872,0.0002133033,0.00003180541,0.0005118911],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.03521487,"threshold_uncertainty_score":0.5693303,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.02389848265672822,"score_gpt":0.2036627314506916,"score_spread":0.1797642487939634,"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."}}