{"id":"W3183781959","doi":"10.1016/j.ymgme.2021.07.006","title":"Gene therapy for Fabry disease: Progress, challenges, and outlooks on gene-editing","year":2021,"lang":"en","type":"review","venue":"Molecular Genetics and Metabolism","topic":"CRISPR and Genetic Engineering","field":"Biochemistry, Genetics and Molecular Biology","cited_by":41,"is_retracted":false,"has_abstract":false,"ca_institutions":"University of Guelph; Dalhousie University","funders":"","keywords":"Genetic enhancement; Genome editing; Gene delivery; Fabry disease; Biology; Disease; Viral vector; Gene; Bioinformatics; Medicine; CRISPR; Genetics; Pathology; 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":[0.0002211289,0.0006853221,0.001057275,0.0001040318,0.0001100072,0.00009157404,0.0002275583,0.0004304222,0.000001984658],"category_scores_gemma":[0.00005362669,0.0006313609,0.0004332624,0.00006354967,0.0001040202,0.000001896887,0.0002309652,0.0001947613,7.381461e-7],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000007593771,"about_ca_system_score_gemma":0.0001476032,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":7.259016e-7,"about_ca_topic_score_gemma":0.000001007312,"domain_scores_codex":[0.997645,0.0001103856,0.0004428505,0.001090088,0.0002180954,0.0004936345],"domain_scores_gemma":[0.9986619,0.00002192374,0.0001660031,0.0007078796,0.0001098226,0.0003324322],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"design_other","study_design_gemma":"not_applicable","study_design_scores_codex":[0.00001096265,0.00004954705,0.000001189658,0.00146125,0.0003664698,0.00002594602,0.00002490725,0.000009614974,0.00161124,0.0003166483,0.000076484,0.9960458],"study_design_scores_gemma":[0.0006561482,0.0001251136,0.00005326728,0.0006238645,0.0006238345,0.00004401003,0.00001484438,0.00002221529,0.007170307,0.00007922954,0.9899151,0.0006720905],"study_design_candidate":"design_other","study_design_consensus":null,"genre_codex":"review","genre_gemma":"review","genre_scores_codex":[0.001324918,0.994554,0.002291061,0.00005789725,0.0004044401,0.001097967,0.0002067474,0.00001914404,0.00004375276],"genre_scores_gemma":[0.0008064876,0.9927561,0.003784466,0.0001440778,0.001022413,0.0004659171,0.0007381353,0.0001857175,0.00009673212],"genre_candidate":"review","genre_consensus":"review","teacher_disagreement_score":0.9953737,"threshold_uncertainty_score":0.9996138,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.02729951381042701,"score_gpt":0.3370344338279793,"score_spread":0.3097349200175523,"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."}}