{"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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0003500419,0.0005640478,0.0004299995,0.0004247309,0.0002717364,0.0005674896,0.0003956791,0.0008370118,0.009892387],"category_scores_gemma":[0.0002131579,0.0002179834,0.0004233364,0.0001765942,0.0005711531,0.0003307756,0.0003281635,0.001512904,0.001370637],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000381583,"about_ca_system_score_gemma":0.0004962514,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.002167996,"about_ca_topic_score_gemma":0.00370062,"domain_scores_codex":[0.9996419,0.00003238029,0.00002812834,0.00009362399,0.0001118677,0.00009203345],"domain_scores_gemma":[0.9998666,0.00003611189,0.0000283744,0.00001494553,0.00001049097,0.00004342188],"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":[0.0002161015,0.0001395889,0.0001201715,0.0001013391,0.00002097783,0.0001155517,0.00003740609,0.00009697396,0.9910424,0.0007153389,0.0007899138,0.006604239],"study_design_scores_gemma":[0.0000713894,0.0004946036,0.001999945,0.00001370795,0.00003071497,0.0004016713,0.00003996639,0.001936967,0.9809822,0.0002172435,0.01380173,0.000009820841],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.8939892,0.004678987,0.06209766,0.002023773,0.002267228,0.0005724531,0.003279423,0.00383944,0.02725183],"genre_scores_gemma":[0.9588371,0.00105877,0.01572976,0.0002556474,0.00004691252,0.0002516148,0.001109398,0.0002128947,0.02249802],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.009892387,"threshold_uncertainty_score":0.03309327,"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."}}