{"id":"W2791875139","doi":"10.1016/j.omtn.2018.03.007","title":"CRISPR/Cas9 Mediated Disruption of the Swedish APP Allele as a Therapeutic Approach for Early-Onset Alzheimer’s Disease","year":2018,"lang":"en","type":"article","venue":"Molecular Therapy — Nucleic Acids","topic":"CRISPR and Genetic Engineering","field":"Biochemistry, Genetics and Molecular Biology","cited_by":189,"is_retracted":false,"has_abstract":true,"ca_institutions":"","funders":"Common Fund; Natural Sciences and Engineering Research Council of Canada; National Cancer Institute; National Institutes of Health; Marcus och Amalia Wallenbergs minnesfond; Hjärnfonden; National Center for Research Resources; National Institute of General Medical Sciences; Kosciuszko Foundation","keywords":"CRISPR; Amyloid precursor protein; Biology; Mutant; Allele; Cas9; Point mutation; Gene; Transgene; Genetics; Molecular biology; Alzheimer's disease; Disease; Medicine; Pathology","routes":{"ca_aff":false,"ca_fund":true,"ca_venue":false,"about_ca":false,"invisible_to_affiliation_only":true},"retraction":null,"screen":null,"direct_labels":[],"prediction":{"model_version":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0002266424,0.0004839404,0.000389962,0.0003612813,0.0002201225,0.0004485785,0.0003520947,0.0005322754,0.002262968],"category_scores_gemma":[0.0001530786,0.0001841245,0.000366367,0.0001484731,0.0002923049,0.0002311882,0.0002794909,0.0008588167,0.0005654845],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0002709621,"about_ca_system_score_gemma":0.000415701,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.000558237,"about_ca_topic_score_gemma":0.001139866,"domain_scores_codex":[0.99983,0.00003037064,0.00002073032,0.00003676864,0.00005737226,0.000024764],"domain_scores_gemma":[0.9999313,0.00001299462,0.0000214201,0.000009500029,0.000007382426,0.00001730235],"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":[0.0001461588,0.00007319512,0.0001198622,0.00008344929,0.00001872344,0.0002100782,0.00001753685,0.0002316548,0.9899025,0.0006982343,0.0003071481,0.008191394],"study_design_scores_gemma":[0.00006157773,0.0004581616,0.001270371,0.00002901761,0.00007969305,0.001684259,0.00002990699,0.001775938,0.9816139,0.0004668244,0.01250905,0.00002139783],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.8583976,0.009976357,0.1089501,0.001284455,0.0008016404,0.0003493891,0.001460846,0.0021418,0.01663788],"genre_scores_gemma":[0.9530373,0.004605733,0.02992405,0.0001967652,0.00003519344,0.0001143672,0.0007705696,0.0001247165,0.01119119],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.002262968,"threshold_uncertainty_score":0.007570446,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.01408906767177368,"score_gpt":0.2980079156000912,"score_spread":0.2839188479283175,"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."}}