{"id":"W2590381489","doi":"10.3390/diseases5010006","title":"A Prospective Treatment Option for Lysosomal Storage Diseases: CRISPR/Cas9 Gene Editing Technology for Mutation Correction in Induced Pluripotent Stem Cells","year":2017,"lang":"en","type":"review","venue":"Diseases","topic":"CRISPR and Genetic Engineering","field":"Biochemistry, Genetics and Molecular Biology","cited_by":15,"is_retracted":false,"has_abstract":true,"ca_institutions":"University of Victoria","funders":"","keywords":"CRISPR; Genome editing; Induced pluripotent stem cell; Cas9; Biology; Computational biology; Genetic enhancement; Homology directed repair; Gene; Bioinformatics; Genetics; DNA repair; Embryonic stem cell; Nucleotide excision repair","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":[0.0002530864,0.0005969682,0.0007508565,0.001996988,0.0002198984,0.0006008596,0.0005305884,0.0008614985,0.002805141],"category_scores_gemma":[0.0002705927,0.0001881959,0.0004758216,0.001336778,0.0002685036,0.0008817368,0.0004588079,0.001499034,0.001497405],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0003589804,"about_ca_system_score_gemma":0.0005493412,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0005041951,"about_ca_topic_score_gemma":0.0009475541,"domain_scores_codex":[0.9998816,0.00001692167,0.00001770653,0.00002203384,0.00005118987,0.00001052033],"domain_scores_gemma":[0.9999003,0.00004257839,0.00001711675,0.000003902228,0.00002528638,0.00001069057],"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":[0.00004360467,0.00006871566,0.0001360653,0.01154407,0.00007878779,0.0003927936,0.00005457038,0.000363157,0.008704595,0.003815692,0.02385613,0.9509416],"study_design_scores_gemma":[0.00001333129,0.00008626898,0.0004715464,0.001556262,0.00008319011,0.002961279,0.00003812442,0.0001215882,0.002627753,0.001518177,0.9905015,0.00002089341],"study_design_candidate":"not_applicable","study_design_consensus":null,"genre_codex":"review","genre_gemma":"review","genre_scores_codex":[0.0002952236,0.9961866,0.0007229341,0.0003664127,0.0002366979,0.000009425098,0.00003495796,0.00002616266,0.002121698],"genre_scores_gemma":[0.001327896,0.9962132,0.0007361958,0.0002573665,0.0001157635,0.00001202322,0.00006950318,0.000003733162,0.001264363],"genre_candidate":"review","genre_consensus":"review","teacher_disagreement_score":0.002805141,"threshold_uncertainty_score":0.009384155,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.03128012698098827,"score_gpt":0.3653439993233172,"score_spread":0.3340638723423289,"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."}}