{"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":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.00006393291,0.0002443717,0.0003382589,0.00003576277,0.00008591409,0.00005192938,0.0001431156,0.00009675919,0.00000583272],"category_scores_gemma":[0.00002729139,0.00016495,0.0001615032,0.00004033717,0.00003115764,0.000001381789,0.000110128,0.00005105125,0.000002870078],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0000049155,"about_ca_system_score_gemma":0.00006043136,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.000004210762,"about_ca_topic_score_gemma":0.000002363399,"domain_scores_codex":[0.9992142,0.00003143613,0.0001539098,0.0003489374,0.00005986206,0.0001916619],"domain_scores_gemma":[0.9994666,0.00007043238,0.00005485864,0.0002881778,0.00002901851,0.00009091675],"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":[0.00001602847,0.00003001141,0.0000363049,0.005714943,0.0002198741,0.000001567069,0.00008411302,0.00001915071,0.001484281,0.000008552845,0.001697178,0.990688],"study_design_scores_gemma":[0.0001633515,0.000113047,0.00002741021,0.0003533181,0.0006729531,0.000008424533,0.0001058096,0.00002839083,0.0006826051,0.000006102483,0.9975712,0.0002673802],"study_design_candidate":"not_applicable","study_design_consensus":null,"genre_codex":"review","genre_gemma":"review","genre_scores_codex":[0.001573133,0.9959186,0.001098627,0.0000338996,0.0001845678,0.0007468702,0.0004233971,0.000009393476,0.00001150503],"genre_scores_gemma":[0.005438999,0.9930125,0.000166344,0.0002246475,0.0006016491,0.0001908668,0.0001646293,0.00004787423,0.0001525304],"genre_candidate":"review","genre_consensus":"review","teacher_disagreement_score":0.995874,"threshold_uncertainty_score":0.6726465,"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."}}