{"id":"W2733851408","doi":"10.15252/emmm.201607137","title":"Orkambi® and amplifier co‐therapy improves function from a rare CFTR mutation in gene‐edited cells and patient tissue","year":2017,"lang":"en","type":"article","venue":"EMBO Molecular Medicine","topic":"Cystic Fibrosis Research Advances","field":"Medicine","cited_by":105,"is_retracted":false,"has_abstract":true,"ca_institutions":"SickKids Foundation; Hospital for Sick Children; University of Toronto","funders":"Hospital for Sick Children; University of Toronto; Canadian Institutes of Health Research; Sick Kids Foundation; Government of Ontario; Cystic Fibrosis Canada; Compute Canada","keywords":"Ivacaftor; Mutation; Cystic fibrosis; Cas9; In silico; Cancer research; CRISPR; Biology; Medicine; Gene; Cystic fibrosis transmembrane conductance regulator; Genetics","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":[0.0002825279,0.0003740346,0.000411065,0.0002187867,0.0001756226,0.0005382413,0.0002626364,0.0004991024,0.002893995],"category_scores_gemma":[0.0002276635,0.0001269573,0.0002840152,0.0001008895,0.0002613468,0.0002159255,0.000334069,0.0009660991,0.0007817567],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0001969308,"about_ca_system_score_gemma":0.0002031391,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0005252533,"about_ca_topic_score_gemma":0.0008706548,"domain_scores_codex":[0.9997483,0.00004674879,0.00002369443,0.00005067893,0.00008362102,0.00004692898],"domain_scores_gemma":[0.9998409,0.00004479195,0.00003071514,0.00002587681,0.00001978425,0.00003795966],"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":[0.00008853812,0.00004710151,0.00014882,0.00005906861,0.000008880115,0.0001497824,0.00002298865,0.0001145539,0.9973845,0.0000618387,0.0001676274,0.001746237],"study_design_scores_gemma":[0.00003121375,0.0009631807,0.002305002,0.00001464967,0.00003718149,0.0009635169,0.00005725908,0.0008649632,0.9881094,0.00003419833,0.006610072,0.000009342654],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9832687,0.00217043,0.009236614,0.0002740248,0.0001327314,0.0001213036,0.0008856184,0.0005140671,0.003396542],"genre_scores_gemma":[0.9922333,0.0007270428,0.002822917,0.0001195664,0.00001194449,0.00007101728,0.0006519454,0.00005818481,0.003304132],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.002893995,"threshold_uncertainty_score":0.009681344,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.01322552082402667,"score_gpt":0.3084802905376418,"score_spread":0.2952547697136151,"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."}}