{"id":"W4415794162","doi":"10.1161/circ.152.suppl_3.4364195","title":"Abstract 4364195: A transformative LDL cholesterol–lowering <i>in vivo</i> CRISPR gene editing medicine that functionally upregulates LDLR in mice and non-human primates","year":2025,"lang":"en","type":"article","venue":"Circulation","topic":"CRISPR and Genetic Engineering","field":"Biochemistry, Genetics and Molecular Biology","cited_by":1,"is_retracted":false,"has_abstract":true,"ca_institutions":"University of British Columbia","funders":"","keywords":"LDL receptor; Genome editing; Downregulation and upregulation; CRISPR; Familial hypercholesterolemia; Untranslated region; PCSK9; Cas9; Gene","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":[0.0004573316,0.0006315892,0.0004826334,0.0004532347,0.0003003622,0.0005159673,0.0005233884,0.0008646252,0.00399144],"category_scores_gemma":[0.0001892753,0.0002395032,0.0005984637,0.0002055565,0.0004346307,0.0002220784,0.0003494451,0.001178293,0.001466239],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0003398559,"about_ca_system_score_gemma":0.0003624202,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0006434752,"about_ca_topic_score_gemma":0.0008286912,"domain_scores_codex":[0.9995775,0.0000586634,0.00003525974,0.0001145625,0.0001536891,0.00006027547],"domain_scores_gemma":[0.9998604,0.00002203709,0.00004591269,0.00001987116,0.00001600162,0.00003575619],"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":[0.0001082283,0.00005039802,0.0001005364,0.0001219036,0.00001495316,0.000142436,0.00002746374,0.0001236845,0.9937766,0.0006498415,0.0005703018,0.004313782],"study_design_scores_gemma":[0.00007112571,0.0008040479,0.0006852453,0.00002481929,0.00006142417,0.001148754,0.00002640768,0.001258332,0.9677407,0.0001908054,0.0279666,0.00002170381],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.6393393,0.00947142,0.2901413,0.002297465,0.001315607,0.001704928,0.007684559,0.008220167,0.03982525],"genre_scores_gemma":[0.8669873,0.004018208,0.0872876,0.0009395287,0.0001129737,0.0009314637,0.004548948,0.000556886,0.03461703],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.00399144,"threshold_uncertainty_score":0.01335275,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.007069927089927009,"score_gpt":0.2847467637653683,"score_spread":0.2776768366754413,"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."}}