{"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":"codex-gemma-dda1882f352a","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0006348325,0.0001972602,0.0002299698,0.0001705911,0.00006716018,0.00003764864,0.0002447317,0.00004077523,0.0001970307],"category_scores_gemma":[0.0003070387,0.0001939036,0.00008541266,0.0003411873,0.00007729472,0.00007645325,0.00003985213,0.00008388473,0.00001849288],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.00003628766,"about_ca_system_score_gemma":0.00005867889,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0000442347,"about_ca_topic_score_gemma":0.000008962568,"domain_scores_codex":[0.997191,0.0009412564,0.0003489726,0.0005327102,0.0006865359,0.0002995069],"domain_scores_gemma":[0.9990852,0.00008764481,0.0001654992,0.0004677165,0.0001252718,0.00006863722],"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":[0.00004438629,0.0001035521,0.0002238018,0.000004832877,0.000004111361,0.00001873776,0.0002598295,0.005847271,0.9897342,0.0002846578,0.00002752123,0.003447106],"study_design_scores_gemma":[0.001263894,0.0001010347,0.002308949,0.00001464634,0.00000863547,0.000006714256,0.00001981916,0.007080749,0.9881034,0.0005548908,0.0003527286,0.0001845137],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9977898,0.0003253761,0.0007669288,0.000118045,0.0001473197,0.0004397566,0.00001102211,0.00002665792,0.0003750906],"genre_scores_gemma":[0.9985176,0.0002779751,0.0002586179,0.0008236596,0.00003891383,0.00003018795,0.000001943398,0.00004022401,0.00001083693],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.003262592,"threshold_uncertainty_score":0.7907158,"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."}}