{"id":"W2999353727","doi":"10.1016/j.ymthe.2019.12.012","title":"Gene Therapy for Glaucoma by Ciliary Body Aquaporin 1 Disruption Using CRISPR-Cas9","year":2020,"lang":"en","type":"article","venue":"Molecular Therapy","topic":"Retinal Development and Disorders","field":"Biochemistry, Genetics and Molecular Biology","cited_by":94,"is_retracted":false,"has_abstract":true,"ca_institutions":"","funders":"UCL Institute of Ophthalmology, University College London; Medical Research Council; Elizabeth Blackwell Institute for Health Research, University of Bristol; Moorfields Eye Hospital NHS Foundation Trust; NIHR Biomedical Research Centre, Royal Marsden NHS Foundation Trust/Institute of Cancer Research; Queen's University; Sight Research UK; University of Bristol; National Institute for Health and Care Research; Queen's University Belfast; Retina UK; Fight for Sight","keywords":"CRISPR; Glaucoma; Genetic enhancement; Ciliary body; Biology; Cas9; Gene; Genome editing; Cell biology; Ophthalmology; Genetics; Medicine; Neuroscience","routes":{"ca_aff":false,"ca_fund":true,"ca_venue":false,"about_ca":false,"invisible_to_affiliation_only":true},"retraction":null,"screen":null,"direct_labels":[],"prediction":{"model_version":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0001183167,0.0002559793,0.0001843972,0.00002629758,0.0001072168,0.000045308,0.0002238613,0.0001566471,0.00003263925],"category_scores_gemma":[0.00002224469,0.0002438832,0.0001876739,0.0001134554,0.0000464227,0.000006813024,0.00003755022,0.00006778281,0.000005975148],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00001259917,"about_ca_system_score_gemma":0.00006302952,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00001290139,"about_ca_topic_score_gemma":6.539179e-7,"domain_scores_codex":[0.998732,0.00007004871,0.0002295106,0.000482969,0.0001724854,0.0003129438],"domain_scores_gemma":[0.999469,0.000006750162,0.00008459907,0.0002442637,0.00007023353,0.0001250835],"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":[0.0007313217,0.00007730546,0.002364986,0.000008628938,0.0001073398,0.000005638405,0.00007528815,0.00001164511,0.9857165,0.00002569612,0.002742821,0.008132798],"study_design_scores_gemma":[0.002025796,0.0005635126,0.0007018773,0.000003693504,0.000008331345,0.000004535159,0.000036808,0.0004029378,0.8759342,0.0001405037,0.1198445,0.0003332503],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9098494,0.006221801,0.08215607,0.0009178966,0.00009788119,0.000570227,0.00002567435,0.00002861362,0.0001324324],"genre_scores_gemma":[0.9878536,0.002591079,0.003190095,0.005086565,0.0001716034,0.00007538634,0.0008452403,0.0000587568,0.0001276942],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.1171017,"threshold_uncertainty_score":0.994527,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.02016380694827731,"score_gpt":0.2767374120487039,"score_spread":0.2565736051004266,"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."}}