{"id":"W2323582185","doi":"10.1038/mtna.2016.7","title":"Design, Characterization, and Lead Selection of Therapeutic miRNAs Targeting Huntingtin for Development of Gene Therapy for Huntington's Disease","year":2016,"lang":"en","type":"article","venue":"Molecular Therapy — Nucleic Acids","topic":"RNA Research and Splicing","field":"Biochemistry, Genetics and Molecular Biology","cited_by":132,"is_retracted":false,"has_abstract":true,"ca_institutions":"University of British Columbia","funders":"","keywords":"Huntingtin; Gene silencing; Gene knockdown; Exon; Huntington's disease; Biology; Exon skipping; microRNA; RNA interference; Transgene; Genetics; Gene; Molecular biology; RNA; Mutant; Medicine; Alternative splicing; Disease","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":[0.0002939871,0.0002916274,0.0003371536,0.0001313981,0.0001307916,0.0003647853,0.0001840508,0.0002737822,0.0003195093],"category_scores_gemma":[0.0003483215,0.000156729,0.0002285877,0.0001123893,0.0001665067,0.0001330904,0.0001517092,0.0003489211,0.0003022331],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0002760447,"about_ca_system_score_gemma":0.0002811505,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0002109291,"about_ca_topic_score_gemma":0.0004588906,"domain_scores_codex":[0.9997973,0.00002677218,0.0000227229,0.00004122598,0.00009124011,0.00002066262],"domain_scores_gemma":[0.9998589,0.00002060659,0.00005096853,0.00001488963,0.00003001814,0.00002453935],"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":[0.00004720768,0.00001944826,0.0001377521,0.00003623873,0.000005395336,0.00004092752,0.0000266934,0.0004962573,0.9948688,0.0002156921,0.00003767742,0.004068097],"study_design_scores_gemma":[0.00001117947,0.0003265382,0.0004825398,0.00000278497,0.00001375639,0.0001183292,0.000007123019,0.001402564,0.9947987,0.00004719084,0.002784256,0.000005157794],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9382576,0.003442757,0.0541492,0.0001410279,0.0000563452,0.0004739516,0.0004693531,0.0002114458,0.002798325],"genre_scores_gemma":[0.9352527,0.002103478,0.05823231,0.00007545062,0.00001594648,0.0002289083,0.0007459076,0.00007162846,0.003273731],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.0003647853,"threshold_uncertainty_score":0.002002835,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.01804486005384942,"score_gpt":0.2661811289913967,"score_spread":0.2481362689375473,"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."}}