{"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":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0005177413,0.0001877385,0.0002150506,0.00007550614,0.0001386633,0.00001910395,0.0001689282,0.00009997263,0.000009203797],"category_scores_gemma":[0.00009291243,0.0001432361,0.0001094184,0.00009207464,0.00008031571,0.00001142958,0.00002759786,0.00003251465,3.936019e-7],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00001603021,"about_ca_system_score_gemma":0.0001392121,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.000002224279,"about_ca_topic_score_gemma":6.265993e-7,"domain_scores_codex":[0.9987341,0.00008434106,0.0003549982,0.0003529894,0.0001604083,0.0003131251],"domain_scores_gemma":[0.9991612,0.00004151465,0.0002190299,0.0001897916,0.0003102632,0.00007818596],"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":[0.001203627,0.00007599367,0.002787687,0.0000413807,0.0001431887,1.171982e-7,0.00008658262,0.000003728536,0.8861852,0.00003039418,0.000009630642,0.1094325],"study_design_scores_gemma":[0.002066517,0.0005319819,0.005514735,0.0000337709,0.000009623888,5.306673e-7,0.00001485705,0.0002277705,0.9843075,0.00005869339,0.007049948,0.0001840592],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.5659825,0.0004919051,0.4328297,0.0001002576,0.00001940991,0.0005571445,0.00001008627,0.000007487652,0.00000147192],"genre_scores_gemma":[0.968679,0.0004888892,0.03006295,0.0001690948,0.00006570821,0.0001762217,0.00008741246,0.00006137235,0.0002093157],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.4027668,"threshold_uncertainty_score":0.5840998,"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."}}