{"id":"W3024777633","doi":"10.1016/j.omtm.2020.05.002","title":"Allele-Specific Prevention of Nonsense-Mediated Decay in Cystic Fibrosis Using Homology-Independent Genome Editing","year":2020,"lang":"en","type":"article","venue":"Molecular Therapy — Methods & Clinical Development","topic":"Cystic Fibrosis Research Advances","field":"Medicine","cited_by":39,"is_retracted":false,"has_abstract":true,"ca_institutions":"Hospital for Sick Children; University of Toronto","funders":"Canadian Institutes of Health Research; Hospital for Sick Children; Cystic Fibrosis Canada","keywords":"Cystic fibrosis; Nonsense; Allele; Genetics; Genome editing; Genome; Homology (biology); Biology; Computational biology; Evolutionary biology; Gene","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":[0.0005457537,0.0004645475,0.0003744156,0.0004106833,0.0002938389,0.0004818203,0.0004888778,0.0008311317,0.001994588],"category_scores_gemma":[0.0003764952,0.0001813569,0.0004585316,0.0002122983,0.0005832682,0.0002852363,0.0004716224,0.001212756,0.0004933202],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0002173033,"about_ca_system_score_gemma":0.0003574684,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0004279893,"about_ca_topic_score_gemma":0.0006267178,"domain_scores_codex":[0.9996443,0.00007892405,0.00002926254,0.00009484752,0.0001043363,0.00004820695],"domain_scores_gemma":[0.9998279,0.00004302474,0.00004171099,0.00004745606,0.00001322202,0.00002668561],"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":[0.0001063659,0.000065443,0.0001165019,0.00008850326,0.00002358279,0.0001679397,0.00003684941,0.0002814949,0.9863034,0.001672673,0.0002937949,0.01084336],"study_design_scores_gemma":[0.00005036216,0.000494134,0.0006640519,0.0000182032,0.00005051672,0.0009187109,0.00001448684,0.002213041,0.982381,0.0003809998,0.0127981,0.00001641842],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.7485805,0.007434281,0.2241481,0.001049251,0.000723317,0.0008290735,0.001305128,0.002747797,0.01318266],"genre_scores_gemma":[0.965956,0.001865299,0.02388083,0.0001910383,0.00003712899,0.0001470204,0.0006832784,0.0001923207,0.007047092],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.001994588,"threshold_uncertainty_score":0.006672561,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.1402007187031849,"score_gpt":0.4556116660790647,"score_spread":0.3154109473758798,"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."}}