{"id":"W4403611477","doi":"10.3390/genes15101342","title":"Recent Progress of Antisense Oligonucleotide Therapy for Superoxide-Dismutase-1-Mutated Amyotrophic Lateral Sclerosis: Focus on Tofersen","year":2024,"lang":"en","type":"review","venue":"Genes","topic":"Amyotrophic Lateral Sclerosis Research","field":"Medicine","cited_by":12,"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":"Amyotrophic lateral sclerosis; Superoxide dismutase; Oligonucleotide; SOD1; Biology; Medicine; Cancer research; Virology; Genetics; Gene; Disease; Pathology; Biochemistry; Enzyme","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":[0.0008512261,0.0006423505,0.0008545825,0.0009319098,0.0001505119,0.0006924081,0.0005019275,0.000739536,0.002087557],"category_scores_gemma":[0.0006262389,0.0001953509,0.000666459,0.000590049,0.000321683,0.0009301849,0.0004367389,0.001661878,0.0007325495],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0003399759,"about_ca_system_score_gemma":0.0005677755,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0004548322,"about_ca_topic_score_gemma":0.0008209254,"domain_scores_codex":[0.9997693,0.00005115465,0.00003576732,0.00005406595,0.00006407395,0.00002565274],"domain_scores_gemma":[0.9997076,0.0001500846,0.00003647501,0.000007673138,0.00006998798,0.00002820057],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"design_other","study_design_gemma":"not_applicable","study_design_scores_codex":[0.0004942641,0.0003133065,0.0005234113,0.01400019,0.0002557377,0.000552017,0.0002559283,0.0007033995,0.02790456,0.005693539,0.02134094,0.9279627],"study_design_scores_gemma":[0.0002059003,0.001767441,0.001717669,0.001627364,0.0005612795,0.002465248,0.0001181971,0.0005666298,0.01397575,0.002406125,0.9745247,0.00006372562],"study_design_candidate":"not_applicable","study_design_consensus":null,"genre_codex":"review","genre_gemma":"review","genre_scores_codex":[0.002282265,0.993509,0.0012834,0.0005588642,0.0004370793,0.00001838126,0.0000417711,0.00003210634,0.001837105],"genre_scores_gemma":[0.009963804,0.985204,0.001463101,0.001018371,0.0005829249,0.00003994875,0.0001617056,0.00001507275,0.001551161],"genre_candidate":"review","genre_consensus":"review","teacher_disagreement_score":0.002087557,"threshold_uncertainty_score":0.006983578,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.1370977705371468,"score_gpt":0.3884359873114527,"score_spread":0.2513382167743059,"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."}}