{"id":"W2517855006","doi":"10.1016/j.nmd.2016.06.444","title":"Gene Therapy 2.0: CRISPR/Cas9 genome editing provides novel therapeutic avenues for neuromuscular disorders","year":2016,"lang":"en","type":"article","venue":"Neuromuscular Disorders","topic":"CRISPR and Genetic Engineering","field":"Biochemistry, Genetics and Molecular Biology","cited_by":2,"is_retracted":false,"has_abstract":false,"ca_institutions":"Hospital for Sick Children; University of Toronto; SickKids Foundation","funders":"","keywords":"CRISPR; Genome editing; Cas9; Utrophin; Duchenne muscular dystrophy; Biology; Gene duplication; Computational biology; Genetic enhancement; Gene; Dystrophin; Bioinformatics; Genetics","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":[0.0006526,0.0006527549,0.0006879144,0.0007582942,0.0004315651,0.001219303,0.0007402886,0.00125633,0.003637436],"category_scores_gemma":[0.000394282,0.0003334479,0.0006234317,0.0003215003,0.0007413165,0.0006869406,0.0007287074,0.001659569,0.001010522],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0006450517,"about_ca_system_score_gemma":0.0007441619,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0008801831,"about_ca_topic_score_gemma":0.001549405,"domain_scores_codex":[0.9994665,0.00006831699,0.00004257656,0.0001340447,0.0002200488,0.00006862218],"domain_scores_gemma":[0.9998417,0.00004234379,0.00004658687,0.00002023911,0.00001849062,0.00003058201],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"bench_or_experimental","study_design_gemma":"theoretical_or_conceptual","study_design_scores_codex":[0.0001880265,0.00007172921,0.0003163942,0.0002671744,0.00004385522,0.0003139445,0.00005971225,0.0005628837,0.9613327,0.005306849,0.001960294,0.02957652],"study_design_scores_gemma":[0.0000905084,0.0004405887,0.002067683,0.00008335258,0.0001258773,0.003019818,0.0000540167,0.004478931,0.9315217,0.002151601,0.05590755,0.00005843278],"study_design_candidate":"theoretical_or_conceptual","study_design_consensus":null,"genre_codex":"methods","genre_gemma":"review","genre_scores_codex":[0.4320965,0.03973499,0.4533909,0.008189008,0.001755442,0.0006084034,0.003115982,0.008893806,0.05221498],"genre_scores_gemma":[0.823989,0.01867502,0.1207414,0.001287093,0.0002344245,0.0002918855,0.001595107,0.0006965026,0.03248955],"genre_candidate":"review","genre_consensus":null,"teacher_disagreement_score":0.003637436,"threshold_uncertainty_score":0.01216841,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.01083753495435886,"score_gpt":0.2698534123215801,"score_spread":0.2590158773672212,"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."}}