{"id":"W4390262237","doi":"10.1016/j.jns.2023.121093","title":"Development of a gene therapy treatment for RYR1-related diseases using prime editing","year":2023,"lang":"en","type":"article","venue":"Journal of the Neurological Sciences","topic":"Virus-based gene therapy research","field":"Biochemistry, Genetics and Molecular Biology","cited_by":0,"is_retracted":false,"has_abstract":false,"ca_institutions":"Université Laval; Centre hospitalier de l'Université Laval","funders":"","keywords":"Genetic enhancement; Genome editing; RYR1; Prime (order theory); Gene; Biology; Genetics; Computational biology; Medicine; CRISPR; Mathematics; Receptor; Ryanodine receptor","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":[0.0003381315,0.0004774928,0.000402182,0.0003086545,0.0002273729,0.0004485396,0.0005054298,0.0004897423,0.002085731],"category_scores_gemma":[0.0001755334,0.0001776018,0.0005128811,0.0001182854,0.0002724937,0.0004087665,0.0002566927,0.001081813,0.0003971624],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0002116051,"about_ca_system_score_gemma":0.0003549965,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0003405254,"about_ca_topic_score_gemma":0.0003348013,"domain_scores_codex":[0.9998852,0.00001807157,0.00000656242,0.00004020567,0.00002883598,0.00002116267],"domain_scores_gemma":[0.9999356,0.00001815368,0.00001363206,0.000009119301,0.00000927257,0.00001426316],"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":[0.0002280278,0.0001347733,0.0001222303,0.0001181386,0.0000212904,0.0001316424,0.00003851728,0.0003266814,0.9766223,0.001854783,0.0002203112,0.0201813],"study_design_scores_gemma":[0.0001858869,0.002073596,0.0007458152,0.00002534687,0.0001096814,0.0009227378,0.00002335163,0.002374725,0.9731631,0.0005538801,0.01980207,0.00001995204],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.7515635,0.0155342,0.2144332,0.001295529,0.0006240327,0.001051888,0.0006525976,0.001214575,0.01363052],"genre_scores_gemma":[0.8949751,0.0076698,0.08502732,0.0003368352,0.00007817069,0.0003994768,0.0006574661,0.0001343415,0.01072139],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.002085731,"threshold_uncertainty_score":0.006977499,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.09077624075167559,"score_gpt":0.3632411633320506,"score_spread":0.272464922580375,"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."}}