{"id":"W2994781405","doi":"10.1016/j.jcf.2019.12.001","title":"Functional rescue of c.3846G&gt;A (W1282X) in patient-derived nasal cultures achieved by inhibition of nonsense mediated decay and protein modulators with complementary mechanisms of action","year":2019,"lang":"en","type":"article","venue":"Journal of Cystic Fibrosis","topic":"Cystic Fibrosis Research Advances","field":"Medicine","cited_by":70,"is_retracted":false,"has_abstract":true,"ca_institutions":"SickKids Foundation; Hospital for Sick Children; University of Toronto","funders":"Cystic Fibrosis Canada; Canadian Institutes of Health Research; Hospital for Sick Children; Cystic Fibrosis Foundation Therapeutics","keywords":"Medicine; Cystic fibrosis; Nonsense; Action (physics); Pharmacology; Internal medicine; Genetics; 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":[0.0002707294,0.0004477322,0.0004759668,0.0001568277,0.0002279726,0.0002765892,0.0003203423,0.0005516475,0.00180689],"category_scores_gemma":[0.000203704,0.0001587944,0.0004595183,0.0001379285,0.000224846,0.0001272681,0.0002933158,0.0007356913,0.0008642652],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0002150146,"about_ca_system_score_gemma":0.0002335869,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.000764497,"about_ca_topic_score_gemma":0.0009860548,"domain_scores_codex":[0.9997541,0.00003722159,0.00004112886,0.00005092427,0.00008508284,0.00003155234],"domain_scores_gemma":[0.9997999,0.00004667306,0.00004726053,0.00003670947,0.00001966964,0.00004974006],"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":[0.00003137402,0.00001395702,0.0001078877,0.00001219281,0.000002683633,0.0001231169,0.00001035898,0.00002029406,0.9993017,0.00001383449,0.00002094502,0.0003416874],"study_design_scores_gemma":[0.00002424761,0.0005047597,0.002567719,0.000005577467,0.00001756151,0.001613893,0.00003496899,0.0006311735,0.9928325,0.00001263265,0.001750754,0.000004229849],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9962249,0.0004945604,0.001770354,0.0001022038,0.00003673934,0.00006940398,0.0005716341,0.0000695843,0.0006608089],"genre_scores_gemma":[0.9918406,0.0005193339,0.0041788,0.00005803163,0.00001213119,0.00008959429,0.001171123,0.00005026623,0.002079993],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.00180689,"threshold_uncertainty_score":0.006044626,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.01050388718971888,"score_gpt":0.2579854291886858,"score_spread":0.247481541998967,"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."}}