{"id":"W7139232359","doi":"","title":"Assessing a Novel Dual-Endonuclease System in Restoring CFTR Expression in Cystic Fibrosis Models","year":2025,"lang":"","type":"dissertation","venue":"TSpace (University of Toronto)","topic":"CRISPR and Genetic Engineering","field":"Biochemistry, Genetics and Molecular Biology","cited_by":0,"is_retracted":false,"has_abstract":true,"ca_institutions":"University of Toronto","funders":"","keywords":"Cystic fibrosis transmembrane conductance regulator; Cystic fibrosis; Chloride channel; Genetic enhancement; Mutation; Lung; Electroporation; Regulator","routes":{"ca_aff":true,"ca_fund":false,"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":[0.0006455339,0.0005766092,0.0004841019,0.000418855,0.0002334593,0.0006128682,0.0004686981,0.0008587603,0.001577806],"category_scores_gemma":[0.0002893567,0.0002112387,0.0003958239,0.000182952,0.000347276,0.0003144535,0.0003241205,0.0009461651,0.0004183309],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0006606407,"about_ca_system_score_gemma":0.0005492663,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00179669,"about_ca_topic_score_gemma":0.003447403,"domain_scores_codex":[0.999532,0.00006703311,0.00003404805,0.000124579,0.0001632197,0.00007908516],"domain_scores_gemma":[0.9997589,0.00004067455,0.00005877598,0.00002386065,0.00005150515,0.00006633769],"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":[0.0001560824,0.0001236743,0.00008889246,0.00003504355,0.000007050341,0.00001389051,0.0000134914,0.00008453356,0.9973236,0.0000501023,0.00006049205,0.00204308],"study_design_scores_gemma":[0.00004839812,0.004049841,0.001207154,0.00001125003,0.00004195097,0.00008783808,0.00002010684,0.0007730296,0.9921773,0.00002329383,0.001552854,0.000006996871],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9685136,0.002179938,0.02490404,0.0003229536,0.0001069114,0.0004103952,0.0007590477,0.0002978913,0.002505242],"genre_scores_gemma":[0.9403654,0.003511145,0.04081585,0.0001944435,0.000027772,0.0007036535,0.001527374,0.000111553,0.01274273],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.00179669,"threshold_uncertainty_score":0.00527823,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.0139878857589207,"score_gpt":0.2812753707104207,"score_spread":0.2672874849515001,"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."}}