{"id":"W3095116115","doi":"10.3390/jpm10040209","title":"Preclinical Studies of a Rare CF-Causing Mutation in the Second Nucleotide Binding Domain (c.3700A>G) Show Robust Functional Rescue in Primary Nasal Cultures by Novel CFTR Modulators","year":2020,"lang":"en","type":"article","venue":"Journal of Personalized Medicine","topic":"Cystic Fibrosis Research Advances","field":"Medicine","cited_by":28,"is_retracted":false,"has_abstract":true,"ca_institutions":"Hospital for Sick Children; University of Toronto","funders":"Government of Canada","keywords":"Potentiator; Cystic fibrosis transmembrane conductance regulator; Cystic fibrosis; Mutation; Compound heterozygosity; HEK 293 cells; Chloride channel; Chemistry; Medicine; Molecular biology; Cancer research; Biology; Gene; Pharmacology; Genetics; Cell 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":[0.0004471099,0.0005746093,0.0008250731,0.0002428519,0.0003388324,0.0003210357,0.0004507279,0.0007483724,0.00237064],"category_scores_gemma":[0.0001786351,0.0001941698,0.0005488529,0.0001438099,0.0004260241,0.0002797148,0.0001783424,0.001625132,0.0007624759],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0003660219,"about_ca_system_score_gemma":0.0004997822,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.001226649,"about_ca_topic_score_gemma":0.002651399,"domain_scores_codex":[0.9997321,0.00005215791,0.00002318682,0.00004263761,0.000079708,0.00007018005],"domain_scores_gemma":[0.9997856,0.00003248643,0.00004602485,0.00002729021,0.00002826238,0.00008038084],"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":[0.0008903735,0.0006745588,0.0002264123,0.00007275359,0.00002495834,0.0001697551,0.00002999932,0.0001507938,0.993964,0.0000988711,0.0003190317,0.00337853],"study_design_scores_gemma":[0.0007746188,0.03877907,0.005241964,0.00002755484,0.000162887,0.00127651,0.00008853598,0.0008152273,0.9439702,0.0001056561,0.008737038,0.00002080209],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.99222,0.00141379,0.001647749,0.0003854698,0.0001373453,0.0003855299,0.001167933,0.0001670477,0.002475151],"genre_scores_gemma":[0.9908042,0.001445851,0.002102103,0.0002043107,0.00002693679,0.0003159314,0.001405961,0.00005302249,0.003641627],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.00237064,"threshold_uncertainty_score":0.007930517,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.09079052436716303,"score_gpt":0.3732188309015349,"score_spread":0.2824283065343719,"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."}}