{"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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0003545698,0.0007795945,0.001034984,0.001168536,0.0001701245,0.0006656246,0.0006949032,0.0008258038,0.002484565],"category_scores_gemma":[0.0003274716,0.0001872805,0.0003753772,0.0009425724,0.0003128007,0.0004659522,0.0004807772,0.001621634,0.00138169],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0005015372,"about_ca_system_score_gemma":0.000658919,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.001058573,"about_ca_topic_score_gemma":0.002296809,"domain_scores_codex":[0.9998909,0.00001504513,0.00001241102,0.00001861376,0.00004699453,0.0000160518],"domain_scores_gemma":[0.9999101,0.0000436193,0.00001269798,0.000003662719,0.00001863387,0.00001123747],"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":[0.00008892246,0.00006070703,0.00004771029,0.008175816,0.00007044637,0.0001857958,0.00001942807,0.0004517578,0.004587379,0.002634611,0.03184142,0.9518361],"study_design_scores_gemma":[0.00004818259,0.000104409,0.0004961155,0.002020869,0.0001662822,0.001347271,0.00001897808,0.0001751825,0.002533098,0.001730984,0.9913345,0.0000240781],"study_design_candidate":"not_applicable","study_design_consensus":null,"genre_codex":"review","genre_gemma":"review","genre_scores_codex":[0.0001970693,0.9973075,0.0005500237,0.0002416645,0.0002472433,0.000006954612,0.00003531049,0.00002593506,0.001388362],"genre_scores_gemma":[0.001650082,0.995791,0.0005020426,0.000233938,0.0001101933,0.00001169287,0.0000708025,0.000004762132,0.00162545],"genre_candidate":"review","genre_consensus":"review","teacher_disagreement_score":0.002484565,"threshold_uncertainty_score":0.008311689,"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."}}