{"id":"W2042183771","doi":"10.1038/mtm.2013.5","title":"Targeted CNS delivery using human MiniPromoters and demonstrated compatibility with adeno-associated viral vectors","year":2014,"lang":"en","type":"article","venue":"Molecular Therapy — Methods & Clinical Development","topic":"CRISPR and Genetic Engineering","field":"Biochemistry, Genetics and Molecular Biology","cited_by":57,"is_retracted":false,"has_abstract":true,"ca_institutions":"Canada's Michael Smith Genome Sciences Centre; Child and Family Research Institute; University of British Columbia","funders":"National Institute of General Medical Sciences; National Eye Institute; National Institutes of Health; Genome British Columbia; Research to Prevent Blindness; Child and Family Research Institute; Michael Smith Health Research BC; Macula Vision Research Foundation; GlaxoSmithKline; Canadian Institutes of Health Research; Genome Canada","keywords":"Biology; Adeno-associated virus; Gene; Gene expression; Reporter gene; Viral vector; Genetic enhancement; Vectors in gene therapy; Locus (genetics); Genetics; Neuroscience; 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":[0.0004528789,0.0004235755,0.0002841024,0.0002513384,0.0001596875,0.0004592295,0.0002770329,0.0003406982,0.001522279],"category_scores_gemma":[0.0003654569,0.0001843365,0.0002911376,0.0001513125,0.0003746735,0.0002267716,0.000265958,0.0006977441,0.0007002958],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0002911435,"about_ca_system_score_gemma":0.0004168635,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0005315566,"about_ca_topic_score_gemma":0.0007053767,"domain_scores_codex":[0.9997352,0.00005041963,0.00002488758,0.00006675198,0.00008675153,0.00003600484],"domain_scores_gemma":[0.9998191,0.00004631542,0.00004830656,0.0000310145,0.00002986774,0.00002539314],"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":[0.00005676995,0.00001564081,0.0001158454,0.0000451607,0.000005124897,0.00005835969,0.00003474871,0.0001401531,0.9946084,0.0004914105,0.00007947522,0.004348985],"study_design_scores_gemma":[0.000008697401,0.0001742966,0.0003860334,0.00000735835,0.000009397509,0.0003208279,0.000009508964,0.0004856715,0.9923038,0.00007549163,0.00621455,0.000004329366],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.7251354,0.00452838,0.2590629,0.0005424154,0.0001680875,0.0008730555,0.001647585,0.0009879268,0.007054267],"genre_scores_gemma":[0.8582866,0.00447763,0.1234602,0.0001710959,0.00005011231,0.0004920352,0.002135578,0.0002175528,0.01070915],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.001522279,"threshold_uncertainty_score":0.005092502,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.04121332399426751,"score_gpt":0.4150032049627303,"score_spread":0.3737898809684628,"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."}}