{"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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0004818545,0.001000604,0.0003426937,0.000635412,0.0002297162,0.000364919,0.0005422209,0.0009257204,0.003974563],"category_scores_gemma":[0.0002730351,0.0003932515,0.0005108806,0.0003784779,0.0002803312,0.0003486789,0.0003454188,0.001261845,0.003107938],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0005278526,"about_ca_system_score_gemma":0.0004436101,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0003813852,"about_ca_topic_score_gemma":0.0006758154,"domain_scores_codex":[0.9994512,0.00009774634,0.00006279046,0.0001157271,0.0001993392,0.00007319242],"domain_scores_gemma":[0.9998156,0.00002954699,0.00006399526,0.00003065201,0.00003024707,0.00002987282],"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":[0.0001046832,0.00006836973,0.0001187712,0.000196892,0.00001754029,0.0001455422,0.00003119127,0.0001792616,0.9845732,0.0008922888,0.001251215,0.01242105],"study_design_scores_gemma":[0.00009833038,0.0003421492,0.001038483,0.00006000558,0.00006337574,0.001734848,0.00001367502,0.001720498,0.9246991,0.0001633188,0.07004718,0.00001914816],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.4817312,0.02862655,0.4279011,0.002086646,0.002438423,0.005023151,0.01087376,0.005580004,0.03573928],"genre_scores_gemma":[0.6020476,0.01576007,0.2867211,0.0009120643,0.0002318391,0.004235097,0.01891349,0.001229234,0.06994959],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.003974563,"threshold_uncertainty_score":0.01329625,"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."}}