{"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":"codex-gemma-dda1882f352a","candidate_categories":["metaepi_narrow"],"consensus_categories":[],"category_scores_codex":[0.0001043689,0.0002691443,0.000172487,0.00006417467,0.00005982325,0.00007553386,0.0003134802,0.0001211044,0.0000449387],"category_scores_gemma":[0.000008212663,0.0002472262,0.0001013616,0.0001560855,0.00004527351,0.000004498965,0.00007567434,0.00006546664,0.00007312844],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00001441171,"about_ca_system_score_gemma":0.0001358257,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00001925366,"about_ca_topic_score_gemma":0.000004410604,"domain_scores_codex":[0.998702,0.00006591967,0.0001782277,0.0004752931,0.0002345309,0.000343967],"domain_scores_gemma":[0.9991699,0.000002674999,0.00003810987,0.0004701474,0.00008377793,0.0002353157],"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":[0.0001702424,0.0001125869,0.0001346372,0.000001676428,0.00005254687,0.00004601825,0.0001720461,0.0008917017,0.989587,0.00004210023,0.002999628,0.005789796],"study_design_scores_gemma":[0.001836927,0.001543372,0.003387099,0.000003754608,0.000004068875,0.00004602301,0.0001703548,0.000007498338,0.8626706,0.0006206332,0.1292752,0.000434492],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9882142,0.004094836,0.002532613,0.0003961953,0.0001318009,0.0003747843,0.00001195836,0.00002931881,0.004214295],"genre_scores_gemma":[0.9923936,0.001046463,0.001442425,0.003346752,0.00002274533,0.00007606307,0.0001087649,0.00006444771,0.001498713],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.1269164,"threshold_uncertainty_score":0.999998,"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."}}