{"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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0002631242,0.0002938283,0.0002944651,0.0001842734,0.0001135587,0.0003167116,0.0002461655,0.0002957433,0.0009733573],"category_scores_gemma":[0.0002093269,0.0001065923,0.0002153351,0.00009751862,0.0002978826,0.0001808526,0.0001928158,0.0005101196,0.0003427835],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0002981958,"about_ca_system_score_gemma":0.0002739804,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0004809464,"about_ca_topic_score_gemma":0.0008877208,"domain_scores_codex":[0.9997342,0.00003692161,0.00001933801,0.0000634646,0.000105848,0.00004026825],"domain_scores_gemma":[0.9998629,0.00003279861,0.00005279363,0.00001846987,0.00001217951,0.00002089775],"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":[0.00001582534,0.00001035658,0.00003696709,0.00001840444,0.000001644654,0.0000274697,0.000005558223,0.00005541771,0.9983012,0.00008510637,0.00001351902,0.001428497],"study_design_scores_gemma":[0.000005247868,0.000132531,0.0005191616,0.000001952293,0.000005436547,0.0001574236,0.000004374634,0.000482791,0.9975402,0.00002666908,0.001122055,0.00000216121],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9283404,0.002814066,0.06385554,0.0002067473,0.0000993103,0.0001483335,0.0003107625,0.000447377,0.00377756],"genre_scores_gemma":[0.9738714,0.001279088,0.01980498,0.00005848649,0.00002552886,0.00004501217,0.0002911621,0.00005647289,0.004567739],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.0009733573,"threshold_uncertainty_score":0.003256142,"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."}}