{"id":"W2057758054","doi":"10.1038/mt.2014.153","title":"In Vivo Evaluation of Candidate Allele-specific Mutant Huntingtin Gene Silencing Antisense Oligonucleotides","year":2014,"lang":"en","type":"article","venue":"Molecular Therapy","topic":"Genetic Neurodegenerative Diseases","field":"Neuroscience","cited_by":148,"is_retracted":false,"has_abstract":true,"ca_institutions":"Child and Family Research Institute; University of British Columbia","funders":"Michael Smith Health Research BC; Canadian Institutes of Health Research; Ionis Pharmaceuticals; Huntington Society of Canada","keywords":"Huntingtin; Huntington's disease; Gene silencing; Polyglutamine tract; Biology; Allele; Trinucleotide repeat expansion; Huntingtin Protein; Single-nucleotide polymorphism; Genetics; Disease; Mutant; Medicine; Gene; Internal medicine; Genotype","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":[0.0003022239,0.0004866192,0.0003295788,0.0002258692,0.0001101727,0.0002127615,0.0002160778,0.000331386,0.0006061245],"category_scores_gemma":[0.0001423641,0.0001247635,0.0001837716,0.00009984564,0.0002076996,0.000151941,0.00006721385,0.0004215467,0.0001175231],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000192983,"about_ca_system_score_gemma":0.0001853459,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0006477701,"about_ca_topic_score_gemma":0.001231056,"domain_scores_codex":[0.9998881,0.00002472867,0.000008610415,0.00003196303,0.00002430406,0.00002237397],"domain_scores_gemma":[0.9998769,0.00003815131,0.00003454387,0.000008270234,0.00002024221,0.00002183389],"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":[0.0001282182,0.0001136204,0.00004391013,0.00003496137,0.000007167928,0.00001383725,0.00001523697,0.000180984,0.998024,0.00005758518,0.00002739082,0.001353216],"study_design_scores_gemma":[0.00004243071,0.004869439,0.0004017349,0.000003899697,0.00003438438,0.00004812438,0.00001462724,0.0007700169,0.9931526,0.00002371347,0.0006357579,0.000003314488],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9893242,0.001432883,0.007574018,0.00006076296,0.00006248776,0.0001496228,0.0002097923,0.00009543178,0.00109079],"genre_scores_gemma":[0.9863281,0.002379029,0.007990208,0.0000490894,0.00002360453,0.0001768578,0.0003098043,0.00002635072,0.002716962],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.0006477701,"threshold_uncertainty_score":0.00202769,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.03340718256104357,"score_gpt":0.2811476605642021,"score_spread":0.2477404780031585,"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."}}