{"id":"W2921219680","doi":"10.1016/j.ymthe.2019.03.003","title":"Non-viral Delivery of Zinc Finger Nuclease mRNA Enables Highly Efficient In Vivo Genome Editing of Multiple Therapeutic Gene Targets","year":2019,"lang":"en","type":"article","venue":"Molecular Therapy","topic":"CRISPR and Genetic Engineering","field":"Biochemistry, Genetics and Molecular Biology","cited_by":94,"is_retracted":false,"has_abstract":true,"ca_institutions":"Acuitas Therapeutics (Canada)","funders":"Sangamo Therapeutics","keywords":"Zinc finger nuclease; Genome editing; Transgene; Biology; Zinc finger; Gene; Gene delivery; Genetic enhancement; In vivo; Genome; Messenger RNA; Gene knockout; Computational biology; Cell biology; Genetics","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":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0001630821,0.0001941076,0.0002532891,0.00009477621,0.00001947331,0.000007605994,0.0002136838,0.000123387,0.00004005819],"category_scores_gemma":[0.000009682519,0.0001917057,0.0001501047,0.000105774,0.00004308116,0.000002739093,0.00006768767,0.00007858132,0.000004389472],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00001435331,"about_ca_system_score_gemma":0.00003851885,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.00006001986,"about_ca_topic_score_gemma":0.000004956179,"domain_scores_codex":[0.9988824,0.0000363638,0.0003146103,0.0003114863,0.0001701796,0.0002850059],"domain_scores_gemma":[0.9993671,0.00001215194,0.0001055813,0.0003928805,0.00007245456,0.00004980757],"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":[0.0001150303,0.0001241649,0.002057537,0.00003957068,0.00007565758,0.000006790919,0.0001848039,0.04168711,0.9548372,0.000008682531,0.000007560381,0.0008558655],"study_design_scores_gemma":[0.001372486,0.0002768858,0.003533578,0.00002292372,0.00000858671,0.000002684307,0.00005813781,0.003607082,0.989226,0.000009651645,0.001687278,0.0001947431],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9886901,0.006368035,0.004293617,0.00001873528,0.0001452489,0.0003113377,0.00002158543,0.000007479337,0.000143906],"genre_scores_gemma":[0.9984247,0.0003015028,0.0009437365,0.0001250311,0.00006626578,0.00001206294,0.00002809941,0.0000442198,0.00005438521],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.03808003,"threshold_uncertainty_score":0.7817531,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.004535572124586177,"score_gpt":0.2267766480793731,"score_spread":0.222241075954787,"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."}}