{"id":"W2563564231","doi":"10.1016/s1525-0016(16)33621-8","title":"17. Gene Therapy Strategies to Treat Fragile X Syndrome","year":2015,"lang":"en","type":"article","venue":"Molecular Therapy","topic":"Genetics and Neurodevelopmental Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":0,"is_retracted":false,"has_abstract":true,"ca_institutions":"University of Toronto","funders":"","keywords":"FMR1; Fragile X syndrome; Transgene; Biology; Gene isoform; Adeno-associated virus; Autism spectrum disorder; Vector (molecular biology); Woodchuck hepatitis virus; Phenotype; Genetic enhancement; Genetically modified mouse; Gene; Viral vector; Genetics; Virology; Autism; Medicine; Fragile x; Virus; Recombinant DNA","routes":{"ca_aff":true,"ca_fund":false,"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":[0.0001423467,0.0003795953,0.0002613488,0.0004830205,0.0001900611,0.0003478336,0.000259013,0.0005271527,0.008063292],"category_scores_gemma":[0.00007929441,0.00007713374,0.000334697,0.0001999681,0.0001873853,0.0002332063,0.0002420599,0.0008145075,0.00288638],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0002696431,"about_ca_system_score_gemma":0.0002180463,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0005452995,"about_ca_topic_score_gemma":0.00122701,"domain_scores_codex":[0.9999479,0.000007130112,0.000003975653,0.000009797634,0.000019299,0.00001188636],"domain_scores_gemma":[0.9999759,0.000002593331,0.000007008783,0.000001914546,0.000005487486,0.000007043182],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"design_other","study_design_gemma":"theoretical_or_conceptual","study_design_scores_codex":[0.0005167479,0.000574994,0.0005222566,0.002219839,0.0001101774,0.001320608,0.0001545267,0.0006205628,0.4339539,0.02052796,0.03688862,0.5025898],"study_design_scores_gemma":[0.0003389576,0.002284449,0.00271101,0.0004799832,0.0001638521,0.003829253,0.0000709104,0.0006191782,0.1329115,0.003092427,0.8534688,0.00002969598],"study_design_candidate":"theoretical_or_conceptual","study_design_consensus":null,"genre_codex":"review","genre_gemma":"methods","genre_scores_codex":[0.1337616,0.6264494,0.04536833,0.01072665,0.008978643,0.0009719645,0.003833197,0.001760808,0.1681495],"genre_scores_gemma":[0.3650025,0.4445013,0.02634656,0.005833732,0.001650523,0.001254686,0.003732944,0.0002260241,0.1514518],"genre_candidate":"methods","genre_consensus":null,"teacher_disagreement_score":0.008063292,"threshold_uncertainty_score":0.02697438,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.02004161643411385,"score_gpt":0.2548312577261461,"score_spread":0.2347896412920323,"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."}}