{"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":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.002620932,0.0002482767,0.001091399,0.0003659285,0.00007656947,0.00001487773,0.0001901137,0.0001132811,0.0002769923],"category_scores_gemma":[0.006028283,0.0001488003,0.0001889238,0.000772457,0.000638961,0.0002903467,0.00005923621,0.001022717,0.000001116546],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0003640654,"about_ca_system_score_gemma":0.0003541241,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00001640738,"about_ca_topic_score_gemma":0.00006160373,"domain_scores_codex":[0.9958936,0.0003004799,0.001401135,0.0003027987,0.001755545,0.0003464536],"domain_scores_gemma":[0.9971017,0.00151186,0.0005587976,0.0001457978,0.0004352048,0.0002466785],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"bench_or_experimental","study_design_gemma":"observational","study_design_scores_codex":[0.01192851,0.0007520604,0.01737738,0.001912163,0.0007090035,0.001107613,0.03237114,0.001220454,0.8953448,0.0001273345,0.03572611,0.001423413],"study_design_scores_gemma":[0.1485694,0.01729234,0.3847921,0.02489021,0.001369579,0.006249804,0.3610276,0.0078921,0.008779027,0.001073132,0.03668761,0.00137698],"study_design_candidate":"bench_or_experimental","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9677073,0.009866664,0.001021124,0.02052168,0.0002173176,0.0004875313,0.00003431686,0.000009072097,0.0001349603],"genre_scores_gemma":[0.9849908,0.0005291986,0.01073154,0.002354644,0.001085809,0.00001504417,0.00005790901,0.00003817125,0.0001968884],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.8865658,"threshold_uncertainty_score":0.7216854,"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."}}