{"id":"W2954109914","doi":"10.3390/diseases7030047","title":"Cell and Gene Therapies for Mucopolysaccharidoses: Base Editing and Therapeutic Delivery to the CNS","year":2019,"lang":"en","type":"review","venue":"Diseases","topic":"CRISPR and Genetic Engineering","field":"Biochemistry, Genetics and Molecular Biology","cited_by":14,"is_retracted":false,"has_abstract":true,"ca_institutions":"University of Victoria","funders":"Rare Disease Foundation; BC Children's Hospital; Children's Hospital Foundation","keywords":"Genome editing; CRISPR; Computational biology; Genetic enhancement; Genome; Gene; Biology; Disease; RNA editing; Neurodegeneration; Neuroscience; Bioinformatics; Medicine; Genetics; RNA; Pathology","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":[0.0003298372,0.0007625293,0.0009607212,0.002091399,0.0002189641,0.0006271561,0.0005845448,0.0008968227,0.00290338],"category_scores_gemma":[0.0003593243,0.0002147581,0.0003682461,0.001591334,0.000314927,0.0007872836,0.0005110866,0.001344164,0.001909865],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0004046639,"about_ca_system_score_gemma":0.000575786,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0005309901,"about_ca_topic_score_gemma":0.0009492591,"domain_scores_codex":[0.9998524,0.00002186107,0.00001985297,0.0000254743,0.00006513613,0.00001522142],"domain_scores_gemma":[0.9998753,0.00006644064,0.00001632665,0.000005161856,0.00002404185,0.00001260838],"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":[0.0000431942,0.00007539491,0.00009359384,0.01125869,0.00006748979,0.0002995854,0.00005602383,0.0004045994,0.006194201,0.003930712,0.02004928,0.9575272],"study_design_scores_gemma":[0.00001443427,0.0001063466,0.0004985289,0.001786162,0.0000723455,0.002154856,0.00003754545,0.0001053309,0.001782844,0.001478908,0.9919459,0.00001662911],"study_design_candidate":"not_applicable","study_design_consensus":null,"genre_codex":"review","genre_gemma":"review","genre_scores_codex":[0.0002450133,0.9971541,0.0003742315,0.0001620363,0.0002258419,0.00000837766,0.00002038761,0.00001478641,0.001795243],"genre_scores_gemma":[0.0008772512,0.9974161,0.0003758209,0.0001308583,0.00009162104,0.00001058757,0.00003685399,0.000002600636,0.001058344],"genre_candidate":"review","genre_consensus":"review","teacher_disagreement_score":0.00290338,"threshold_uncertainty_score":0.009712756,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.02153632507444079,"score_gpt":0.3192491955485723,"score_spread":0.2977128704741315,"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."}}