{"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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.001160572,0.0009397549,0.001805992,0.001343368,0.0002000968,0.001243701,0.001203205,0.001461982,0.002882977],"category_scores_gemma":[0.0008431801,0.000243126,0.0005024492,0.001325494,0.0006762352,0.001347446,0.0007478922,0.002673898,0.001614446],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0006748761,"about_ca_system_score_gemma":0.0009285613,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0007059756,"about_ca_topic_score_gemma":0.001553253,"domain_scores_codex":[0.9997746,0.00004565234,0.00002087308,0.00003736447,0.00008724537,0.0000342253],"domain_scores_gemma":[0.9995759,0.000272833,0.00003546269,0.00001386445,0.00006463539,0.00003731798],"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":[0.0001276295,0.00007319819,0.00008229894,0.01096578,0.00008919793,0.0002024477,0.00003926942,0.000555378,0.003588243,0.008674869,0.03621824,0.9393836],"study_design_scores_gemma":[0.00003845527,0.00009762275,0.0003026334,0.002441528,0.0001056108,0.0008941072,0.0000341097,0.000139194,0.001180983,0.003756216,0.9909876,0.00002194947],"study_design_candidate":"not_applicable","study_design_consensus":null,"genre_codex":"review","genre_gemma":"review","genre_scores_codex":[0.00005862911,0.9987675,0.0002578159,0.000284689,0.0001710201,0.000002071451,0.00001063226,0.00000897134,0.0004388208],"genre_scores_gemma":[0.0004466682,0.9985496,0.0002765009,0.0002027012,0.0001129212,0.000004271252,0.00002107889,0.000002643266,0.0003835605],"genre_candidate":"review","genre_consensus":"review","teacher_disagreement_score":0.002882977,"threshold_uncertainty_score":0.009644568,"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."}}