{"id":"W2922072440","doi":"10.1042/etls20180059","title":"Development of therapeutic genome engineering in laminin-α2-deficient congenital muscular dystrophy","year":2019,"lang":"en","type":"article","venue":"Emerging Topics in Life Sciences","topic":"CRISPR and Genetic Engineering","field":"Biochemistry, Genetics and Molecular Biology","cited_by":0,"is_retracted":false,"has_abstract":true,"ca_institutions":"University of Toronto; Hospital for Sick Children","funders":"Hospital for Sick Children; Muscular Dystrophy Association","keywords":"Muscular dystrophy; Congenital muscular dystrophy; Laminin; Genetic enhancement; Cas9; Genome editing; Gene; Bioinformatics; Medicine; Genome; Biology; CRISPR; Genetics; Computational biology; Extracellular matrix","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":[0.0003763375,0.0004081258,0.0004367951,0.0003667647,0.0001489024,0.0005219897,0.0003832518,0.0006967876,0.001289179],"category_scores_gemma":[0.0002209394,0.0001712084,0.0004152301,0.0001818008,0.0002588585,0.0002727999,0.0003810877,0.0008627113,0.0004018197],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0003545355,"about_ca_system_score_gemma":0.0003556125,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0003611277,"about_ca_topic_score_gemma":0.00054442,"domain_scores_codex":[0.9997925,0.00004916757,0.00001812507,0.00003763716,0.00006966309,0.00003295425],"domain_scores_gemma":[0.9999223,0.0000196517,0.00002599027,0.000006648956,0.00001165854,0.0000137225],"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":[0.00008093785,0.00007802193,0.00009240845,0.0006542153,0.00003723231,0.0003197938,0.00005346503,0.0009466832,0.9677483,0.002835189,0.0006837632,0.02646988],"study_design_scores_gemma":[0.00008885044,0.001605234,0.001838425,0.0002745795,0.0001953809,0.001866266,0.0000864175,0.004762786,0.8884317,0.001297892,0.0995158,0.00003659723],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.5916487,0.1066498,0.2558802,0.003141278,0.001563967,0.0009031688,0.001807372,0.002704365,0.03570126],"genre_scores_gemma":[0.8410373,0.05786309,0.08806742,0.0006521967,0.00008520481,0.0004772763,0.001257269,0.0002347247,0.01032556],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.001289179,"threshold_uncertainty_score":0.004312754,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.01137900794908383,"score_gpt":0.278268971272976,"score_spread":0.2668899633238922,"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."}}