{"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":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0001431085,0.0002185972,0.0002703173,0.00007673681,0.0007564204,0.00003170296,0.0001617125,0.0001097267,0.0003069982],"category_scores_gemma":[0.00000901869,0.0001252077,0.0002496593,0.0002448586,0.0002354821,0.00004137612,0.00001854132,0.0002537478,0.000003439559],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000146879,"about_ca_system_score_gemma":0.0001177233,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.000007626533,"about_ca_topic_score_gemma":0.000005049179,"domain_scores_codex":[0.9985993,0.0001597754,0.0003725301,0.0003023222,0.000291068,0.0002750289],"domain_scores_gemma":[0.9990385,0.0001746484,0.0001750524,0.0004390113,0.00009810203,0.00007464438],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"bench_or_experimental","study_design_gemma":"not_applicable","study_design_scores_codex":[0.00208276,0.003792501,0.0085474,0.0001950059,0.006650978,0.00001241996,0.0931889,0.0004307365,0.4595856,0.002775865,0.0009100653,0.4218278],"study_design_scores_gemma":[0.02869524,0.0139877,0.1091324,0.0001353745,0.008838595,0.0005383565,0.04099189,0.07052209,0.09240303,0.001855951,0.6315899,0.001309415],"study_design_candidate":"not_applicable","study_design_consensus":null,"genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9822413,0.00394701,0.0007338235,0.008682072,0.0001529026,0.003630378,0.0002323538,0.00007633624,0.0003038653],"genre_scores_gemma":[0.9963933,0.001009397,0.0001101002,0.0004755252,0.00008434899,0.0009132185,0.0001435388,0.00003845078,0.0008321736],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.6306799,"threshold_uncertainty_score":0.5817851,"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."}}