{"id":"W4399322906","doi":"10.3390/molecules29112658","title":"Potential of Cell-Penetrating Peptide-Conjugated Antisense Oligonucleotides for the Treatment of SMA","year":2024,"lang":"en","type":"article","venue":"Molecules","topic":"Neurogenetic and Muscular Disorders Research","field":"Medicine","cited_by":9,"is_retracted":false,"has_abstract":true,"ca_institutions":"Muscular Dystrophy Canada; University of Alberta","funders":"Faculty of Medicine and Dentistry, University of Alberta; Canadian Institutes of Health Research; Muscular Dystrophy Canada; University of Alberta; Alberta Innovates - Health Solutions; Women and Children's Health Research Institute; Children's Health Research Institute; Alberta Innovates; Heart and Stroke Foundation of Canada; U.S. Department of Defense","keywords":"SMN1; SMA*; Spinal muscular atrophy; RNA splicing; Oligonucleotide; Alternative splicing; Medicine; Motor neuron; Cancer research; Cell; Morpholino; Gene; Cell biology; RNA; Biology; Chemistry; Bioinformatics; Exon; Pathology; Disease; Biochemistry; Zebrafish; Computer science","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":[0.00006772943,0.00009350691,0.0001719105,0.00006610452,0.000041414,0.00001392012,0.00005512522,0.00003154248,0.00003675548],"category_scores_gemma":[0.00002495569,0.00005836101,0.000203983,0.0001145382,0.0001052708,0.00001013653,0.00001287626,0.00003767944,0.000004191866],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.000009803976,"about_ca_system_score_gemma":0.00008036631,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0000662473,"about_ca_topic_score_gemma":0.000006857035,"domain_scores_codex":[0.9992929,0.00002636187,0.0001919284,0.0001606986,0.0001735956,0.0001544905],"domain_scores_gemma":[0.9995548,0.0001070578,0.00003035173,0.0001924915,0.00007498732,0.00004038215],"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":[0.0001086518,0.0002187184,0.0002890737,0.0003537486,0.0001888166,0.00006128532,0.0002943061,0.00007454304,0.9783232,0.0001697943,0.0002429635,0.01967488],"study_design_scores_gemma":[0.001088523,0.001416391,0.005569076,0.00008565497,0.0002963024,0.00002538936,0.0003427375,0.00790851,0.9805897,0.00006981434,0.002543608,0.0000642682],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.9892363,0.007040496,0.001946798,0.000349875,0.00007447637,0.0006496893,0.00002235382,0.0000202362,0.0006597587],"genre_scores_gemma":[0.9969817,0.00122333,0.001139434,0.0000254727,0.00003849732,0.00002941828,0.00001260987,0.00002388527,0.0005256436],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.01961061,"threshold_uncertainty_score":0.2379893,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.01851531935937214,"score_gpt":0.2942066935427727,"score_spread":0.2756913741834006,"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."}}