{"id":"W3185877187","doi":"10.1016/j.omtm.2021.06.013","title":"Gene therapy using an ortholog of human fragile X mental retardation protein partially rescues behavioral abnormalities and EEG activity","year":2021,"lang":"en","type":"article","venue":"Molecular Therapy — Methods & Clinical Development","topic":"Genetics and Neurodevelopmental Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":31,"is_retracted":false,"has_abstract":true,"ca_institutions":"University of Toronto","funders":"Canadian Institutes of Health Research","keywords":"FMR1; Fragile X syndrome; Biology; Transgene; Gene isoform; Phenotype; Neuroscience; Adeno-associated virus; Genetic enhancement; Genetics; Gene; Fragile x; Vector (molecular biology)","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":[0.0001738681,0.0009371561,0.0004777987,0.0008251267,0.0001879416,0.0002719116,0.0003580804,0.000477328,0.003929585],"category_scores_gemma":[0.0002128522,0.0001905087,0.0003146461,0.0001532575,0.0008967654,0.0003195751,0.0002432783,0.001101972,0.0006535904],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0002251373,"about_ca_system_score_gemma":0.0004147654,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.001525123,"about_ca_topic_score_gemma":0.001575107,"domain_scores_codex":[0.9998667,0.00001770723,0.00001600667,0.00003646214,0.00003399549,0.00002917051],"domain_scores_gemma":[0.9996244,0.00006847755,0.0001217368,0.00004396293,0.00002265073,0.0001187633],"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":[0.000186718,0.00009275432,0.0001584812,0.00003663891,0.00001103181,0.0001279866,0.00002065496,0.00009240901,0.9960935,0.0001937764,0.0001345358,0.002851573],"study_design_scores_gemma":[0.00005862626,0.001173889,0.004436833,0.0000281586,0.00004226277,0.0007844444,0.000105355,0.001070205,0.9888718,0.0002584936,0.003153649,0.00001627916],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9764727,0.001091642,0.01412012,0.0006882609,0.0002417083,0.0001135125,0.00161089,0.001451137,0.004210128],"genre_scores_gemma":[0.9693532,0.002033541,0.009137804,0.0001727162,0.00004056889,0.0002596613,0.001384756,0.0003199571,0.01729794],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.003929585,"threshold_uncertainty_score":0.01314574,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.1304001493316821,"score_gpt":0.4436730021547614,"score_spread":0.3132728528230794,"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."}}