{"id":"W4210599015","doi":"10.1089/nat.2021.0009","title":"Allele-Selective LNA Gapmers for the Treatment of Fibrodysplasia Ossificans Progressiva Knock Down the Pathogenic ACVR1 <sup>R206H</sup> Transcript and Inhibit Osteogenic Differentiation","year":2022,"lang":"en","type":"article","venue":"Nucleic Acid Therapeutics","topic":"Heterotopic Ossification and Related Conditions","field":"Medicine","cited_by":13,"is_retracted":false,"has_abstract":true,"ca_institutions":"Muscular Dystrophy Canada; University of Alberta","funders":"","keywords":"Fibrodysplasia ossificans progressiva; Heterotopic ossification; Biology; Gene knockdown; Myositis ossificans; Molecular biology; Messenger RNA; Genetics; Gene; Cell biology; Medicine; Anatomy; Pathology","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":[0.0001571437,0.0003224476,0.0003973453,0.0002303696,0.0001043773,0.0001943197,0.0002511458,0.0004850523,0.000909471],"category_scores_gemma":[0.000118284,0.0001337788,0.0002769333,0.0001391655,0.0002439047,0.0001473158,0.0001525101,0.0007780371,0.0003134054],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0001518732,"about_ca_system_score_gemma":0.0001420505,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0002073769,"about_ca_topic_score_gemma":0.0004232381,"domain_scores_codex":[0.9998633,0.00002348612,0.00001531643,0.00002773598,0.00004405883,0.0000259652],"domain_scores_gemma":[0.9999477,0.00001242381,0.00001842558,0.000005956078,0.000004921068,0.00001062833],"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":[0.0001972942,0.000104383,0.0000602426,0.00006657954,0.00001101257,0.0001048765,0.00001960985,0.00009622086,0.9932261,0.0001676898,0.0001114633,0.005834583],"study_design_scores_gemma":[0.0001583119,0.002173883,0.0009171231,0.000008947995,0.00005022294,0.0008067098,0.00002261852,0.0007546135,0.9866993,0.00006211158,0.008336648,0.000009506224],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9803149,0.008073217,0.008383877,0.0002708334,0.0001709176,0.0001702733,0.0003636669,0.0003450681,0.001907348],"genre_scores_gemma":[0.9880784,0.002624544,0.005921237,0.0001203522,0.00002361746,0.0001533439,0.0004436464,0.00003467905,0.002600055],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.000909471,"threshold_uncertainty_score":0.003042519,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.02853613819960576,"score_gpt":0.2726857046449064,"score_spread":0.2441495664453007,"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."}}