{"id":"W4390106146","doi":"10.3390/cells13010031","title":"Successful Correction by Prime Editing of a Mutation in the RYR1 Gene Responsible for a Myopathy","year":2023,"lang":"en","type":"article","venue":"Cells","topic":"CRISPR and Genetic Engineering","field":"Biochemistry, Genetics and Molecular Biology","cited_by":15,"is_retracted":false,"has_abstract":true,"ca_institutions":"Université Laval","funders":"Fonds de Recherche du Québec - Santé; Canadian Institutes of Health Research; RYR-1 Foundation","keywords":"RYR1; Mutation; Point mutation; Gene; Genetics; Myopathy; Genome editing; Biology; Ryanodine receptor; Gene mutation; CRISPR; Receptor","routes":{"ca_aff":true,"ca_fund":true,"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":[0.000315649,0.0005289897,0.0005309937,0.0002998184,0.000239303,0.0003478765,0.0003083942,0.000827743,0.001564365],"category_scores_gemma":[0.0005064677,0.0002079335,0.0004524726,0.0001154703,0.0003045674,0.0001386585,0.0004563383,0.001018325,0.0006194737],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0001394971,"about_ca_system_score_gemma":0.0001862788,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0003129426,"about_ca_topic_score_gemma":0.00033689,"domain_scores_codex":[0.9996316,0.00004951773,0.00003611814,0.0001183027,0.0001188764,0.00004552903],"domain_scores_gemma":[0.9996873,0.000116117,0.00008858182,0.00005053081,0.00002135329,0.00003612701],"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":[0.00005548945,0.00002344504,0.0001417277,0.00004875362,0.00001520639,0.0006015762,0.00003885043,0.0002326777,0.9924477,0.0003270647,0.0001259597,0.005941437],"study_design_scores_gemma":[0.00001411357,0.0002372792,0.000938162,0.000009386989,0.00004045478,0.002413634,0.00001322779,0.00158214,0.9868035,0.0001198528,0.007813623,0.00001459924],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.891242,0.001947603,0.09908124,0.0004736755,0.0004218223,0.0002392297,0.0007718644,0.001290967,0.004531602],"genre_scores_gemma":[0.9566141,0.00142095,0.03413167,0.0001593717,0.0000441239,0.0000782556,0.0005982019,0.0003443476,0.006608998],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.001564365,"threshold_uncertainty_score":0.005233288,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.006347292387806518,"score_gpt":0.286491673056758,"score_spread":0.2801443806689515,"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."}}