{"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":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0001796372,0.0004096758,0.0004036,0.0004200622,0.0001067409,0.0002923169,0.0001776414,0.0004356466,0.0007157442],"category_scores_gemma":[0.0001319684,0.0001243639,0.000306956,0.0001927601,0.0001338351,0.0003196967,0.0002054129,0.0005189449,0.0002943402],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0001578472,"about_ca_system_score_gemma":0.0001674411,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0002687248,"about_ca_topic_score_gemma":0.0005240089,"domain_scores_codex":[0.9999195,0.00002063135,0.000007668121,0.00001574008,0.00002303254,0.00001347256],"domain_scores_gemma":[0.9999696,0.000009009755,0.000007007977,0.000001126226,0.000008912068,0.000004243691],"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":[0.0003406983,0.0001775266,0.0002202698,0.003984928,0.0000751996,0.0007213798,0.0001297307,0.0009971893,0.7560462,0.002536766,0.002607256,0.2321629],"study_design_scores_gemma":[0.0002622707,0.006522779,0.003773999,0.0007575509,0.0004780612,0.005190779,0.0001227186,0.003155752,0.5764467,0.001510852,0.4016919,0.00008660876],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"review","genre_gemma":"empirical","genre_scores_codex":[0.143039,0.8248502,0.0173605,0.0009564691,0.000745636,0.0002350568,0.0002291357,0.0002245374,0.01235947],"genre_scores_gemma":[0.3772596,0.5982205,0.0142908,0.000881084,0.0003262115,0.0002447194,0.0003591274,0.00003407582,0.008383863],"genre_candidate":"empirical","genre_consensus":null,"teacher_disagreement_score":0.0007157442,"threshold_uncertainty_score":0.002394378,"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."}}