{"id":"W4213174645","doi":"10.1007/s13311-022-01197-9","title":"CRISPR-Cas9 Gene Therapy for Duchenne Muscular Dystrophy","year":2022,"lang":"en","type":"review","venue":"Neurotherapeutics","topic":"CRISPR and Genetic Engineering","field":"Biochemistry, Genetics and Molecular Biology","cited_by":63,"is_retracted":false,"has_abstract":false,"ca_institutions":"Université Laval","funders":"Fonds de Recherche du Québec - Santé; Canadian Institutes of Health Research","keywords":"CRISPR; Genome editing; Duchenne muscular dystrophy; Cas9; Dystrophin; Computational biology; Genetic enhancement; Biology; Guide RNA; Muscular dystrophy; Gene; Genetics; Bioinformatics; Medicine","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":"codex-gemma-dda1882f352a","candidate_categories":["metaepi_narrow"],"consensus_categories":[],"category_scores_codex":[0.000166451,0.0005812788,0.000809739,0.0001090904,0.0001499283,0.00004924995,0.0005506465,0.00034686,0.0001058398],"category_scores_gemma":[0.00002721842,0.0005489221,0.000892351,0.0001712118,0.00004416016,0.000001767765,0.00009287406,0.00031694,0.00000743725],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0000306581,"about_ca_system_score_gemma":0.0001693191,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.000002474968,"about_ca_topic_score_gemma":9.431155e-7,"domain_scores_codex":[0.9979934,0.0001205762,0.0004624134,0.0007519572,0.0002030087,0.000468698],"domain_scores_gemma":[0.9987156,0.00005548986,0.0001651153,0.0009127323,0.00004781436,0.000103215],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"design_other","study_design_gemma":"not_applicable","study_design_scores_codex":[0.0000358277,0.0001004374,8.282216e-7,0.001611797,0.0004615924,0.000008906895,0.00001822302,0.00009257326,0.004895376,0.00002643087,0.0007372895,0.9920107],"study_design_scores_gemma":[0.0005045741,0.0006077486,9.852617e-7,0.00006890178,0.0002828624,0.00006776605,0.000004946158,0.00002177377,0.002227009,0.0000107804,0.9956681,0.0005345851],"study_design_candidate":"not_applicable","study_design_consensus":null,"genre_codex":"review","genre_gemma":"review","genre_scores_codex":[0.00006308234,0.9865897,0.01063926,0.00001932624,0.0007847082,0.001534629,0.0002190624,0.00004256195,0.0001077085],"genre_scores_gemma":[0.00002703677,0.9951532,0.001274454,0.0004502774,0.0008917121,0.0008119252,0.0005865857,0.0002964784,0.0005083543],"genre_candidate":"review","genre_consensus":"review","teacher_disagreement_score":0.9949308,"threshold_uncertainty_score":0.9996963,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.063210282270666,"score_gpt":0.387382244489429,"score_spread":0.324171962218763,"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."}}